(Q26741955)

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Genome- and Cell-Based Strategies in Therapy of Muscular Dystrophies

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Genome- and Cell-Based Strategies in Therapy of Muscular Dystrophies (English)

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Y Bou Saada

1

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Carla Dib

2

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M Lipinski

3

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Y S Vassetzky

4

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July 2016

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81

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7

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678-90

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Use of Whole-Exome Sequencing for Diagnosis of Limb-Girdle Muscular Dystrophy: Outcomes and Lessons Learned.

1 reference

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7 January 2021

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Evidence-based path to newborn screening for Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

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The ever-expanding spectrum of congenital muscular dystrophies

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

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Oculopharyngeal muscular dystrophy (OPMD) due to a small duplication in the PABPN1 gene

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

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Correction of the FSHD myoblast differentiation defect by fusion with healthy myoblasts

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Endonucleases: tools to correct the dystrophin gene.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

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Ex vivo gene editing of the dystrophin gene in muscle stem cells mediated by peptide nucleic acid single stranded oligodeoxynucleotides induces stable expression of dystrophin in a mouse model for Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

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Analysis of zinc fingers optimized via phage display: evaluating the utility of a recognition code

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

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Immunosuppressive therapy in Duchenne muscular dystrophy: considerations for myoblast transfer studies

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

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Inducing high rates of targeted mutagenesis in zebrafish using zinc finger nucleases (ZFNs).

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Localisation of the myotonic dystrophy locus to 19q13.2-19q13.3 and its relationship to twelve polymorphic loci on 19q

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Mutations in the Caenorhabditis elegans dystrophin-like gene dys-1 lead to hyperactivity and suggest a link with cholinergic transmission

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Minimum length of direct repeat sequences required for efficient homologous recombination induced by zinc finger nuclease in yeast.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Targeted transgene integration in plant cells using designed zinc finger nucleases

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Distal myopathies

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Mapping of facioscapulohumeral muscular dystrophy gene to chromosome 4q35-qter by multipoint linkage analysis and in situ hybridization

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

A direct comparison of the properties of natural and designed zinc-finger proteins

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Congenital muscular dystrophies

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Short telomeres and stem cell exhaustion model Duchenne muscular dystrophy in mdx/mTR mice

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

A long ncRNA links copy number variation to a polycomb/trithorax epigenetic switch in FSHD muscular dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Development and applications of CRISPR-Cas9 for genome engineering

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

The silkworm glutathione S-transferase gene noppera-bo is required for ecdysteroid biosynthesis and larval development.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Low incidence of off-target mutations in individual CRISPR-Cas9 and TALEN targeted human stem cell clones detected by whole-genome sequencing.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Conservation of components of the dystrophin complex in Drosophila.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Inappropriate Gene Activation in FSHD

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Localisation of gene for Becker muscular dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Design, activity, and structure of a highly specific artificial endonuclease

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Fmr1 and Nlgn3 knockout rats: novel tools for investigating autism spectrum disorders

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

The homologue of the Duchenne locus is defective in X-linked muscular dystrophy of dogs

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Structure of the multimodular endonuclease FokI bound to DNA

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Mutations in the gene encoding lamin A/C cause autosomal dominant Emery-Dreifuss muscular dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Analysis of allele-specific RNA transcription in FSHD by RNA-DNA FISH in single myonuclei.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

DNA polymorphism and epigenetic marks modulate the affinity of a scaffold/matrix attachment region to the nuclear matrix

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

De novo mutations in sporadic deletional Duchenne muscular dystrophy (DMD) cases

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

RNA interference improves myopathic phenotypes in mice over-expressing FSHD region gene 1 (FRG1).

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

CRISPR/dCas9-mediated Transcriptional Inhibition Ameliorates the Epigenetic Dysregulation at D4Z4 and Represses DUX4-fl in FSH Muscular Dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Engraftment potential of dermal fibroblasts following in vivo myogenic conversion in immunocompetent dystrophic skeletal muscle

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

DNA interrogation by the CRISPR RNA-guided endonuclease Cas9.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

An improved zinc-finger nuclease architecture for highly specific genome editing

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

A TALE nuclease architecture for efficient genome editing

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Targeted gene disruption in somatic zebrafish cells using engineered TALENs

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Knockout rats generated by embryo microinjection of TALENs.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Replication-dependent instability at (CTG)•(CAG) repeat hairpins in human cells

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

New mdx mutation disrupts expression of muscle and nonmuscle isoforms of dystrophin

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Hypomethylation of D4Z4 in 4q-linked and non-4q-linked facioscapulohumeral muscular dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

CRISPR interference: RNA-directed adaptive immunity in bacteria and archaea

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Evolution and classification of the CRISPR-Cas systems

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Mechanism of nonhomologous end-joining in mycobacteria: a low-fidelity repair system driven by Ku, ligase D and ligase C

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Myoblast Transfer in the Treatment of Duchenne's Muscular Dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Systemic administration of PRO051 in Duchenne's muscular dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Zinc-finger directed double-strand breaks within CAG repeat tracts promote repeat instability in human cells

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

De novo-engineered transcription activator-like effector (TALE) hybrid nuclease with novel DNA binding specificity creates double-strand breaks

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Identification of 86 candidates for small non-messenger RNAs from the archaeon Archaeoglobus fulgidus.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Direct observation of R-loop formation by single RNA-guided Cas9 and Cascade effector complexes.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

X chromosome-linked muscular dystrophy (mdx) in the mouse

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Use of a zinc-finger consensus sequence framework and specificity rules to design specific DNA binding proteins

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Structure of FokI has implications for DNA cleavage

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

FokI dimerization is required for DNA cleavage

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Binding studies with mutants of Zif268. Contribution of individual side chains to binding affinity and specificity in the Zif268 zinc finger-DNA complex

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

The Krüppel-like factor 15 as a molecular link between myogenic factors and a chromosome 4q transcriptional enhancer implicated in facioscapulohumeral dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

A simplified immune suppression scheme leads to persistent micro-dystrophin expression in Duchenne muscular dystrophy dogs

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Myotonic dystrophy kinase is a component of neuromuscular junctions

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Myotilin is mutated in limb girdle muscular dystrophy 1A

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

The zebrafish as a model for muscular dystrophy and congenital myopathy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Strand bias in oligonucleotide-mediated dystrophin gene editing

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Site-directed gene repair of the dystrophin gene mediated by PNA-ssODNs

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Nested chromosomal fragmentation in yeast using the meganuclease I-Sce I: a new method for physical mapping of eukaryotic genomes

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Homing endonucleases: structural and functional insight into the catalysts of intron/intein mobility

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

A combinatorial approach to create artificial homing endonucleases cleaving chosen sequences

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

A nuclear matrix attachment site in the 4q35 locus has an enhancer-blocking activity in vivo: implications for the facio-scapulo-humeral dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Myoblasts from affected and non-affected FSHD muscles exhibit morphological differentiation defects

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Myotonic dystrophy type 2 caused by a CCTG expansion in intron 1 of ZNF9

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

CRISPR Provides Acquired Resistance Against Viruses in Prokaryotes

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

A bacterial effector acts as a plant transcription factor and induces a cell size regulator

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Small CRISPR RNAs guide antiviral defense in prokaryotes

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

CRISPR interference limits horizontal gene transfer in staphylococci by targeting DNA

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Breaking the code of DNA binding specificity of TAL-type III effectors

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

A simple cipher governs DNA recognition by TAL effectors

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Sequence- and Structure-Specific RNA Processing by a CRISPR Endonuclease

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Multiplex genome engineering using CRISPR/Cas systems

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Genome editing. The new frontier of genome engineering with CRISPR-Cas9

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Myotonic dystrophy mutation: an unstable CTG repeat in the 3' untranslated region of the gene

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Gene therapy for human genetic disease?

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Zinc finger-DNA recognition: crystal structure of a Zif268-DNA complex at 2.1 A

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

A Human-Specific Deletion in Mouse Cmah Increases Disease Severity in the mdx Model of Duchenne Muscular Dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Genetic correction of human induced pluripotent stem cells from patients with spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Induction of homologous recombination in mammalian chromosomes by using the I-SceI system of Saccharomyces cerevisiae

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

The role of CRISPR-Cas systems in virulence of pathogenic bacteria

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

FRG2, an FSHD candidate gene, is transcriptionally upregulated in differentiating primary myoblast cultures of FSHD patients

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Expression of the dystrophin-glycoprotein complex is a marker for human airway smooth muscle phenotype maturation

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Function and genetics of dystrophin and dystrophin-related proteins in muscle

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Results of a triple blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Simultaneous miRNA and mRNA transcriptome profiling of human myoblasts reveals a novel set of myogenic differentiation-associated miRNAs and their target genes

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Genetic correction of splice site mutation in purified and enriched myoblasts isolated from mdx5cv mice

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Dynamics of double strand breaks and chromosomal translocations

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Facioscapulohumeral dystrophy: the path to consensus on pathophysiology

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Myotonic dystrophy type 2: molecular, diagnostic and clinical spectrum

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Population-based incidence and prevalence of facioscapulohumeral dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Human umbilical cord blood cells restore brain damage induced changes in rat somatosensory cortex

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

GFP-based fluorescence assay for CAG repeat instability in cultured human cells

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Targeting DNA double-strand breaks with TAL effector nucleases

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Canine X-linked muscular dystrophy in Japan (CXMDJ)

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

Clinical, pathological and genetic characteristics of a pedigree with myotonic dystrophy type 1.

1 reference

https://api.crossref.org/works/10.1134%2FS000629791607004X

7 January 2021

based on heuristic

inferred from DOI database lookup

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10.1134/S000629791607004X

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