Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of ...

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

How CRISPR therapy could cure everything from cancer to infertility The imminent approval of the world's first CRISPR treatment for sickle cell disease is just the start: soon this gene-editing ...

Gene therapy has been available since 2022 for ... “We would call that the low-to-mild range. By definition, there are people that are higher and lower.” Patients must have serious disease ...

The Metachromatic Leukodystrophy Initiative (MLDi) critically responds to the findings of Zhang et al. on lentivirus-modified ...