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Gene therapy for selected neuromuscular and trinucleotide repeat disorders – An insight to subsume South Asia for multicenter clinical trials

Background: In this article, the authors discuss how they utilized the genetic mutation data in Sri Lankan Duchenne muscular dystrophy (DMD), Spinal muscular atrophy (SMA), Spinocerebellar ataxia (SCA) and Huntington’s disease (HD) patients and compare the available literature from South Asian count...

Ausführliche Beschreibung

Gespeichert in:
Hauptverfasser: Nalaka Wijekoon, Lakmal Gonawala, Pyara Ratnayake, Darshana Sirisena, Harsha Gunasekara, Athula Dissanayake, Sunethra Senanayake, Ajantha Keshavaraj, Yetrib Hathout, Harry W.M. Steinbusch, Chandra Mohan, Ashwin Dalal, Eric Hoffman, K.Ranil D de Silva
Format: Artigo
Sprache:Inglês
Veröffentlicht: Elsevier 2023-06-01
Schriftenreihe:IBRO Neuroscience Reports
Schlagworte:
Indian Sub-continent
Developing Countries
Neurogenetic Disorders
Bio Bank
Duchenne Muscular Dystrophy
Spinal Muscular Atrophy
Online-Zugang:http://www.sciencedirect.com/science/article/pii/S266724212300009X
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