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Gene Therapy articles from GeneMedi.
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...
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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...
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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...
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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...
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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...
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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...
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