Knowledge Hub
Gene Therapy articles from GeneMedi.
A preclinical study published in Science Translational Medicine used split intein protein trans-splicing and a muscle-tropic AAV vector to reconstruct full-length dysferlin in an L...
Read article
A 3-year-old child with infantile Krabbe disease developed a GLI1-altered tumor paravertebral to L2–L3 after receiving AAV gene therapy. Long-read sequencing showed that a subgenom...
Read article
ALPK3 gene truncating mutations are an important genetic cause of hypertrophic cardiomyopathy, accounting for approximately 2% of cases. There is currently no curative treatment fo...
Read article
A phase 1 trial in patients aged 12–18 years with severe hemophilia B has yielded an intriguing signal: factor IX activity peaks higher than in adults, but declines more markedly b...
Read article
The combination of suprachoroidal injection and RPE-specific promoters is rewriting the rules of retinal gene delivery. Dry age-related macular degeneration (dAMD) is a leading cau...
Read article
Hereditary cerebellar ataxias have long been regarded as a group of neurodegenerative diseases that are difficult to intervene in. When the causative gene encodes a secreted synapt...
Read article
Sandhoff disease (SD) is caused by defects in the HEXA/HEXB genes, leading to Hex enzyme deficiency and accumulation of GM2 ganglioside in lysosomes. Previous studies mostly delive...
Read article
Adeno-associated virus (AAV) vectors are widely used in gene therapy, but their natural tropism is too broad. This creates a long-standing contradiction: to make them infect only a...
Read articleAbout GeneMedi SOLIDEX®SOLIDEX® is GeneMedi's proprietary comprehensive solution for immune cell solution, dedicated to providing efficient and reliable tools for cell separation a...
Read article
