September 29, 2020

The Niche

Knoepfler lab stem cell blog

Heidi Ledford.

6 min read

Three new studies reported in preprints all show severe DNA damage to human embryos from CRISPR a surprisingly high percentage of the time including in some cases loss of heterozygosity (LOH). Anywhere from about a quarter to half of CRISPR’d embryos exhibited major genome damage. As readers of The Niche know, I never was a fan of the idea of doing CRISPR on human embryos for reproductive use, although I have been supportive of limited, thoughtful in vitro gene-editing studies on human embryos. The …Read More

3 min read

It’s notable how the FDA now considers gene-editing a kind of regenerative medicine. This means that various gene therapy products in development technically qualify as regenerative therapies. Ever since the agency began its rapidly growing regenerative medicine advanced therapy (RMAT) designation program, we’ve seen an increasing number of gene therapy biotechs and products qualify as RMATs. As a result, the RMAT-qualified gene therapies get to go into a speedier lane of FDA oversight. Today’s post is a mix of recommended reads in the regenerative …Read More

2 min read

What can go wrong with unapproved stem cell clinic offerings? The answer including from presentations at the FDA today turns out to be very serious, negative results right here in the U.S. Thomas Albini, MD gave a talk entitled,  “Severe Visual Loss After Intravitreal Injection of Autologous Adipose Tissue-derived Stem Cells for Age-related Macular Degeneration”. Stem cell clinic transplants of fat stem cells led to blindness in three women, reported Dr. Albini. We’ve heard encouraging news about how stem cells might help patients regain …Read More