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Gene Therapy for a Rare Muscular Dystrophy Overcomes Earlier Roadblocks

A preclinical study provides proof-of-concept evidence for a new gene therapy approach that offers a potential long-term treatment for limb-girdle muscular dystrophy 2B. Researchers at Children’s National have developed the first non-muscle targeted gene therapy that enhances diseased muscle fiber repair and improves muscle function through a single low dose of an AAV vector that produces human acid sphingomyelinase, released by muscle cells immediately following muscle injury. ....

United States , Sreetama Sen Chandra , Jyoti Jaiswal , Daniel Bittel , Jyotik Jaiswal , Eric Hoffmann , Bradley Williams , Children National , Genetic Medicine Research At Children National , Binghamton University , Children National Hospital , Jain Foundation , George Washington University School Of Medicine , Health Sciences , National Hospital , Clinical Investigation , Genetic Medicine Research , National Health System , George Washington University School , Sen Chandra ,

Research offers hope for people with limb-girdle muscular dystrophy

Research offers hope for people with limb-girdle muscular dystrophy ....

United States , Daniel Bittel , Jyotik Jaiswal , Us National Library Of Medicine , Genetic Medicine Research At Children National , Us Department Of Health , Human Services , Children National Hospital , National Hospital , Clinical Investigation , Genetic Medicine Research ,

Gut flora composition and function may impact susceptibility to konzo, a neurological disease ...

Gut flora composition and function may impact susceptibility to konzo, a neurological disease ...
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Democratic Republic Of The General , Democratic Republic Of The Congo , Masi Manimba , United States , Bas Congo , Valeria Sabate , Eric Vilain , Matthews Bramble , Neerja Vashist , Institut National De Recherche Biom , Children National , Nature Communications , Genetic Medicine Research At Children National , Valeria Sabate Children National Hospital , Oregon Health Science University , Children National Hospital , Democratic Republic , National Hospital , Genetic Medicine Research , Desire Tshala Katumbay , Oregon Health , Sub Saharan Africa , Sabate Children ,

Gut flora composition and function may impact susceptibility to konzo, neurological disease caused by world staple crop cassava

Gut flora composition and function may impact susceptibility to konzo, neurological disease caused by world staple crop cassava
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Democratic Republic Of The General , Democratic Republic Of The Congo , Masi Manimba , United States , Bas Congo , Eric Vilain , Matthews Bramble , Neerja Vashist , Institut National De Recherche Biom , Children National , Nature Communications , Genetic Medicine Research At Children National , Oregon Health Science University , Children National Hospital , Democratic Republic , National Hospital , Genetic Medicine Research , Desire Tshala Katumbay , Oregon Health , Sub Saharan Africa ,

Gut Flora May Impact Susceptibility to Neurological Disease Konzo, Caused By Cassava Crop

Gut Flora May Impact Susceptibility to Neurological Disease Konzo, Caused By Cassava Crop
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Democratic Republic Of The General , Democratic Republic Of The Congo , Masi Manimba , United States , Bas Congo , Eric Vilain , Matthews Bramble , Neerja Vashist , Institut National De Recherche Biom , Children National , Genetic Medicine Research At Children National , Oregon Health Science University , Children National Hospital , Democratic Republic , National Hospital , Genetic Medicine Research , Nature Genetics , Desire Tshala Katumbay , Oregon Health , Sub Saharan Africa ,