Duchenne muscular dystrophy, one of the most complicated diseases for gene therapy DOI Open Access

Serge Braun

Journal of Translational Genetics and Genomics, Journal Year: 2025, Volume and Issue: 9(1), P. xx - xx

Published: Jan. 25, 2025

Gene therapy for Duchenne muscular dystrophy (DMD) is hindered by many pitfalls related in particular to the limitations of current technologies, specificities muscle and cardiac targets, disease itself, a chronic, multisystem, dystrophic inflammatory disorder. Following RNA-based therapies, DNA gene transfer, mainly based on adeno-associated viral vectors, now able deliver therapeutic genetic sequences massive scale, first antisense virus (AAV)-microdystrophin products are reaching marketing stage Europe and/or US. However, only subset patients eligible those therapies. Many questions remain, such as duration effect, burden high doses immunogenicity capsids proteins, context disease-related background. Evaluations these treatments different biotech, pharma or non-for-profit sponsors also come up against great clinical heterogeneity patients. This review summarizes significant progress made over past three decades optimize both efficacy safety DMD well remaining challenges, short-term prospects, future directions more targeted vectors combination

Language: Английский

Treating neuromuscular diseases: unveiling gene therapy breakthroughs and pioneering future applications DOI Creative Commons
Yu‐Fu Wu, Jun‐An Chen, Yuh‐Jyh Jong

et al.

Journal of Biomedical Science, Journal Year: 2025, Volume and Issue: 32(1)

Published: Feb. 21, 2025

Abstract In this review, we highlight recent advancements in adeno-associated virus (AAV)-based gene therapy for genetic neuromuscular diseases (NMDs), focusing on spinal muscular atrophy (SMA) and Duchenne dystrophy (DMD). We discuss the current FDA-approved therapies NMDs provide updates preclinical studies that demonstrate potential of various AAV-based to reduce SMA severity serve as effective treatments DMD. Additionally, explore transformative impact CRISPR/Cas9 technology future NMDs. Despite these encouraging developments, further research is required identify robust biomarkers can guide treatment decisions predict outcomes. Overall, pioneering lay groundwork efforts aimed at curing offer a roadmap developing other neurodegenerative diseases.

Language: Английский

Citations

2
Duchenne muscular dystrophy, one of the most complicated diseases for gene therapy DOI Open Access

Serge Braun

Journal of Translational Genetics and Genomics, Journal Year: 2025, Volume and Issue: 9(1), P. xx - xx

Published: Jan. 25, 2025

Gene therapy for Duchenne muscular dystrophy (DMD) is hindered by many pitfalls related in particular to the limitations of current technologies, specificities muscle and cardiac targets, disease itself, a chronic, multisystem, dystrophic inflammatory disorder. Following RNA-based therapies, DNA gene transfer, mainly based on adeno-associated viral vectors, now able deliver therapeutic genetic sequences massive scale, first antisense virus (AAV)-microdystrophin products are reaching marketing stage Europe and/or US. However, only subset patients eligible those therapies. Many questions remain, such as duration effect, burden high doses immunogenicity capsids proteins, context disease-related background. Evaluations these treatments different biotech, pharma or non-for-profit sponsors also come up against great clinical heterogeneity patients. This review summarizes significant progress made over past three decades optimize both efficacy safety DMD well remaining challenges, short-term prospects, future directions more targeted vectors combination

Language: Английский

Citations

0