We believe through this therapeutic intervention, we will provide a novel treatment option for ATTR-CM patients, offering them new hope and an improved quality of life.”This study is a part of YolTech’s series of clinical trials in vivo gene editing. mRNA, in the cytoplasm, binds to ribosomes, translating the base editor protein. The base editor protein, in combination with sgRNA, enters the cell nucleus. sgRNA specifically locates the base editor to the TTR gene sequence, and the base editor protein modifies the bases on the target TTR gene, preventing its normal transcription into mRNA. It has created a pipeline with 10+ genetic medicines focusing on cardiovascular diseases, metabolic diseases, infectious diseases as well as more common and rare diseases.

Source: The Bubble December 18, 2023 00:02 UTC