(Q38168863)

30 August 2017

title

New developments in the use of gene therapy to treat Duchenne muscular dystrophy (English)

1 reference

30 August 2017

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Susan Jarmin

1

1 reference

30 August 2017

Hanna Kymalainen

series ordinal

2

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30 August 2017

Linda Popplewell

series ordinal

3

1 reference

30 August 2017

George Dickson

series ordinal

4

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30 August 2017

language of work or name

English

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publication date

6 December 2013

1 reference

Expert Opinion on Biological Therapy

30 August 2017

published in

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30 August 2017

volume

14

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30 August 2017

issue

2

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30 August 2017

page(s)

209-230

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Pharmacologically targeting the primary defect and downstream pathology in Duchenne muscular dystrophy

30 August 2017

cites work

1 reference

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Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient

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A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping

21 January 2018

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Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping

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Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD.

21 January 2018

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Long-term systemic administration of unconjugated morpholino oligomers for therapeutic expression of dystrophin by exon skipping in skeletal muscle: implications for cardiac muscle integrity

21 January 2018

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21 January 2018

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Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice

21 January 2018

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Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles

21 January 2018

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Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells

21 January 2018

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Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy

21 January 2018

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Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials

21 January 2018

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Induced dystrophin exon skipping in human muscle explants

21 January 2018

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Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle

21 January 2018

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Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

21 January 2018

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Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

21 January 2018

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Systemic administration of PRO051 in Duchenne's muscular dystrophy

21 January 2018

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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

21 January 2018

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One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice

21 January 2018

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Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Nanopolymers improve delivery of exon skipping oligonucleotides and concomitant dystrophin expression in skeletal muscle of mdx mice

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Cationic PMMA nanoparticles bind and deliver antisense oligoribonucleotides allowing restoration of dystrophin expression in the mdx mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Formulation of polylactide-co-glycolic acid nanospheres for encapsulation and sustained release of poly(ethylene imine)-poly(ethylene glycol) copolymers complexed to oligonucleotides

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Dystrophin restoration in skeletal, heart and skin arrector pili smooth muscle of mdx mice by ZM2 NP-AON complexes

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Microbubble stability is a major determinant of the efficiency of ultrasound and microbubble mediated in vivo gene transfer.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Exosomes and microvesicles: extracellular vesicles for genetic information transfer and gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Exosome-mediated delivery of siRNA in vitro and in vivo

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Exosomes as nucleic acid nanocarriers

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Cardiomyopathy of Duchenne muscular dystrophy: current understanding and future directions

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Pathophysiology and therapy of cardiac dysfunction in Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

In vivo delivery of naked antisense oligos in aged mdx mice: analysis of dystrophin restoration in skeletal and cardiac muscle.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Cell-penetrating-peptide-based delivery of oligonucleotides: an overview

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Aptamer:toxin conjugates that specifically target prostate tumor cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Selective gene silencing in activated leukocytes by targeting siRNAs to the integrin lymphocyte function-associated antigen-1.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Intracellular small interfering RNA delivery using genetically engineered double-stranded RNA binding protein domain

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Targeted intracellular delivery of antisense oligonucleotides via conjugation with small-molecule ligands.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

RNA aptamer evolution: two decades of SELEction

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Influence of diverse chemical modifications on the ADME characteristics and toxicology of antisense oligonucleotides.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Improved Performance of Anti-miRNA Oligonucleotides Using a Novel Non-Nucleotide Modifier

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Effective exon skipping and dystrophin restoration by 2'-o-methoxyethyl antisense oligonucleotide in dystrophin-deficient mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Antisense properties of tricyclo-DNA

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Nuclear antisense effects in cyclophilin A pre-mRNA splicing by oligonucleotides: a comparison of tricyclo-DNA with LNA

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Improving gene silencing of siRNAs via tricyclo-DNA modification

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

The small molecule Retro-1 enhances the pharmacological actions of antisense and splice switching oligonucleotides.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Identification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screening

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Manipulation of alternative splicing by a newly developed inhibitor of Clks

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Chemical treatment enhances skipping of a mutated exon in the dystrophin gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Readthrough of dystrophin stop codon mutations induced by aminoglycosides

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

PTC124 targets genetic disorders caused by nonsense mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Recombinant adeno-associated virus transduction and integration

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Gene transfer into skeletal muscle using novel AAV serotypes.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Adenovirus and adeno-associated virus as vectors for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Delivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of α1-syntrophin and α-dystrobrevin in skeletal muscles of mdx mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Protein trans-splicing as a means for viral vector-mediated in vivo gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Adeno-associated virus-8-mediated intravenous transfer of myostatin propeptide leads to systemic functional improvements of slow but not fast muscle

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Administration of a soluble activin type IIB receptor promotes skeletal muscle growth independent of fiber type

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Muscle-specific expression of insulin-like growth factor I counters muscle decline in mdx mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Dystrophin immunity in Duchenne's muscular dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

RNAi-mediated knockdown of dystrophin expression in adult mice does not lead to overt muscular dystrophy pathology

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Myostatin propeptide gene delivery by adeno-associated virus serotype 8 vectors enhances muscle growth and ameliorates dystrophic phenotypes in mdx mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Restoration of dystrophin expression using the Sleeping Beauty transposon.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Oligonucleotide-mediated gene repair at DNA level: the potential applications for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Targeted gene correction by small single-stranded oligonucleotides in mammalian cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Gene correction of the apolipoprotein (Apo) E2 phenotype to wild-type ApoE3 by in situ chimeraplasty

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Strand bias in oligonucleotide-mediated dystrophin gene editing

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Correction of dystrophia myotonica type 1 pre-mRNA transcripts by artificial trans-splicing

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Exon exchange approach to repair Duchenne dystrophin transcripts

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Fate of recombinant adeno-associated viral vector genomes during DNA double-strand break-induced gene targeting in human cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Endonucleases: tools to correct the dystrophin gene.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

UtroUp is a novel six zinc finger artificial transcription factor that recognises 18 base pairs of the utrophin promoter and efficiently drives utrophin upregulation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Unexpected failure rates for modular assembly of engineered zinc fingers

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Chimeric nucleases stimulate gene targeting in human cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

DNA-binding specificity is a major determinant of the activity and toxicity of zinc-finger nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Expanding or restricting the target site repertoire of zinc-finger nucleases: the inter-domain linker as a major determinant of target site selectivity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Zinc-finger nucleases: the next generation emerges

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modification

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Xanthomonas AvrBs3 family-type III effectors: discovery and function

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

A TALE nuclease architecture for efficient genome editing

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

TALE nucleases: tailored genome engineering made easy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Targeted Myostatin Gene Editing in Multiple Mammalian Species Directed by a Single Pair of TALE Nucleases.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Optimized TAL effector nucleases (TALENs) for use in treatment of sickle cell disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

TALENs and ZFNs are associated with different mutation signatures

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

A novel engineered meganuclease induces homologous recombination in yeast and mammalian cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Engineered I-CreI derivatives cleaving sequences from the human XPC gene can induce highly efficient gene correction in mammalian cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Meganucleases can restore the reading frame of a mutated dystrophin

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Genetic engineering of human pluripotent cells using TALE nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Optimization of scarless human stem cell genome editing

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Targeted genome modification in mice using zinc-finger nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Gene targeting by homologous recombination in mouse zygotes mediated by zinc-finger nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Correction of the ΔF508 Mutation in the Cystic Fibrosis Transmembrane Conductance Regulator Gene by Zinc-Finger Nuclease Homology-Directed Repair

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Targeted gene knockout by direct delivery of zinc-finger nuclease proteins

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Zinc-finger nucleases for somatic gene therapy: the next frontier

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Antisense-mediated redirection of mRNA splicing

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Antisense oligo-mediated multiple exon skipping in a dog model of duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Multiple exon skipping strategies to by-pass dystrophin mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

Targeted exon skipping to address "leaky" mutations in the dystrophin gene.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2014.866087

10.1517/14712598.2014.866087

21 January 2018

Identifiers

DOI

1 reference

30 August 2017

1 reference