CRISPR gene editing has made its way into every corner of modern biology, but there’s one place CRISPR can’t easily reach: mitochondria. Researchers have been looking for other ways to precisely edit the mitochondrial genome and the past few years have brought some success. Read the article: https://lnkd.in/eMtQXMTc
I think we’re at a “very promising” stage: the barrier (mitochondrial genome editing) that long held back therapies for mtDNA mutation disease is being breached. The advent of mitochondrial base editors is a big leap. If the field can solve delivery and specificity issues, the next decade could see real clinical applications for mitochondrial diseases that currently have very limited treatments.
Not Physical way, Biochemical way preferred
AI/ML/automation architect accelerating drug discovery. Berkeley Chemical Biology × Brandeis Bioinformatics. Ex-Moderna Vaccine QC. Building computational frameworks that cut FDA validation from years to months.
5hRemarkable progress, but the clinical path is long. The key challenge isn't just delivery, but ensuring specificity in an organelle with a high mutation rate and minimal DNA repair. Off-target effects in mtDNA could be uniquely problematic.