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Gene-replacement therapy is helping children overcome fatal diseases

Replacing faulty genes with healthy ones has enabled several children with rare genetic disorders to have typical life experiences, such as attending school and living with their families ....

United States , Donald Kohn , Los Angeles , Alice Klein , University Of California , Rare Diseases , Gene Replacement Therapy , Clinical Trial , Leukocyte Adhesion Deficiency , Mad 1 , Stem Cell , X Linked Severe Combined Immune Deficiency , X Scid , Bubble Boy Disease , M1 Gangliosidosis ,

GC Pharma and Tottori University Enter Research Collaboration and License Agreement for Development of GM1 Gangliosidosis Chaperone Therapy

YONGIN, South Korea, August 02, 2021 GC Pharma (006280.KS) and Tottori University today announced a worldwide licensing and collaboration agreement for the orally administrable chaperone drug of GM1 gangliosidosis (GM1). GM1 is one of the inherited lysosomal storage diseases caused by mutations in the GLB1 gene, that progressively destroys nerve cells in brain and spinal cord. It occurs in approximately 1 out of every 100,000 newborns. There is currently no cure. ....

Tottori University , M1 Gangliosidosis , Lysosomal Storage Diseases , Green Cross Corporation , Eurodegenerative Disease , தோத்டொரி பல்கலைக்கழகம் ,

Sio Gene Therapies Announces Fiscal Year 2020 Year-End Financial Results and Expected Fiscal Year 2021 Key Milestones

Sio Gene Therapies Announces Fiscal Year 2020 Year-End Financial Results and Expected Fiscal Year 2021 Key Milestones
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Gene Therapies , M1 Gangliosidosis , Gene Therapy , Neurodegenerative Diseases , Product Candidates , Sio Gene Therapies , Gene Therapies , Product Development , கீந் சிகிச்சைகள் ,