Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent Landscape

Spinal muscular atrophy (SMA) is a rare autosomal recessive neuromuscular disease that is characterized by progressive muscle atrophy (degeneration), including skeletal muscles in charge of the ability to move. SMA is caused by defects in the SMN1 gene (Survival of Motor Neuron 1) which encodes a pr...

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Detalles Bibliográficos
Autores principales: Ponomarev, Aleksei S., Chulpanova, Daria S., Yanygina, Lina M., Solovyeva, Valeriya V., Rizvanov, Albert A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10530942/
https://www.ncbi.nlm.nih.gov/pubmed/37762045
http://dx.doi.org/10.3390/ijms241813743

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10530942/
https://www.ncbi.nlm.nih.gov/pubmed/37762045
http://dx.doi.org/10.3390/ijms241813743

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