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[HTML][HTML] Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy

A Pickar-Oliver, V Gough, JD Bohning, S Liu… - Molecular Therapy, 2021 - cell.com
Targeted gene-editing strategies have emerged as promising therapeutic approaches for
the permanent treatment of inherited genetic diseases. However, precise gene correction …
被引用次数：37 相关文章

Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.

A Pickar-Oliver, V Gough, JD Bohning… - … Therapy: the Journal …, 2021 - europepmc.org
Targeted gene-editing strategies have emerged as promising therapeutic approaches for
the permanent treatment of inherited genetic diseases. However, precise gene correction …
[HTML] sciencedirect.com

[HTML][HTML] Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy

A Pickar-Oliver, V Gough, JD Bohning, S Liu… - Molecular Therapy, 2021 - Elsevier
Targeted gene-editing strategies have emerged as promising therapeutic approaches for
the permanent treatment of inherited genetic diseases. However, precise gene correction …
[HTML] nih.gov

[HTML][HTML] Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy

A Pickar-Oliver, V Gough, JD Bohning, S Liu… - Molecular …, 2021 - ncbi.nlm.nih.gov
Targeted gene-editing strategies have emerged as promising therapeutic approaches for
the permanent treatment of inherited genetic diseases. However, precise gene correction …
[HTML] cell.com

[HTML][HTML] Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy

A Pickar-Oliver, V Gough, JD Bohning, S Liu… - Molecular Therapy, 2021 - cell.com
Targeted gene-editing strategies have emerged as promising therapeutic approaches for
the permanent treatment of inherited genetic diseases. However, precise gene correction …

Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy

A Pickar-Oliver, V Gough… - … therapy: the journal …, 2021 - pubmed.ncbi.nlm.nih.gov
Targeted gene-editing strategies have emerged as promising therapeutic approaches for
the permanent treatment of inherited genetic diseases. However, precise gene correction …