Stoke Therapeutics - Autosomal Dominant Optic Atrophy (ADOA) скачать в хорошем качестве

Stoke Therapeutics - Autosomal Dominant Optic Atrophy (ADOA)

3D science and Mode of Action animation to explain Stoke's ADOA therapy and TANGO platform What is TANGO? TANGO stands for Targeted Augmentation of Nuclear Gene Output, and it's a proprietary research platform developed by Stoke. It's primarily used to treat diseases caused by mutated genes that don't produce enough protein, called haploinsufficiences. TANGO utilizes RNA science and antisense oligonucleotides (ASOs) to increase the protein output from healthy genes and compensate for the missing protein from the mutated gene. How TANGO Works TANGO focuses on genetic diseases where the patient has one healthy and one mutated gene, leading to a reduction in protein production by half. The gene's transcription into pre-messenger RNA (pre-mRNA) happens next. The pre-mRNA can either be processed into productive mRNA, which is used to produce protein, or non-productive mRNA. Both types of mRNA can exist within the cell. The non-productive mRNA is degraded and doesn't result in protein production. Only the productive mRNA from the healthy gene generates the necessary protein. Stoke Science in Action Stoke synthesizes small modified RNA fragments called antisense oligonucleotides (ASOs) that target specific regions of pre-mRNA. Preclinical studies have shown that ASO therapy reduces non-productive mRNA and increases productive mRNA, leading to increased protein production from the healthy gene. As a result, the target protein is restored to near normal levels. Stoke's ASOs have been proven to reduce non-productive mRNA and increase both productive mRNA and protein levels in genes of varying size, type, and function in a dose-dependent manner.