Drugs that restore the shape of the errant protein behind cystic fibrosis (CF) have, over the past eight years, helped the majority of patients, who have certain mutations. Gene-corrected stem cells might offer a “mutation agnostic” option to CF. CF results from a glitch in a glycoprotein with the unwieldy name “cystic fibrosis transmembrane conductance […]
Can Gene-Edited Stem Cells Treat Cystic Fibrosis? Read More »
Something called, “The International Commission on the Clinical Use of Human Germline Genome Editing” has issued a new report on heritable human genome editing (HHGE). The Commission was convened by the U.S. National Academy of Medicine, the U.S. National Academy of Sciences, and the U.K.’s Royal Society. This post is my initial take on the summary of
New human genome editing (HHGE) academy report: solid, but key gaps Read More »
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
Regenerative medicine recommended reads including $191 million to researcher Read More »
When I say “gene edit” or “genome editing”, what’s the first thing that pops in your mind? It will depend on who you are. For many lay people until a few weeks ago when the world heard about He Jiankui‘s claim of CRISPR’d babies they may not have had anything pop in their heads when
Context for my Nature piece on redefining “gene edit” to be more precise Read More »
Did Chinese researcher He Jiankui really CRISPR gene edit the CCR5 gene in two embryos producing twin baby girls? In my opinion the answer is “no”, but probably not for the reason you might think at first. He proclaims gene edits He claimed he had made twin baby girls with “gene edits”, which I feel is unethical
He Jiankui didn’t really gene edit those girls; he mutated them Read More »
It’s an odd confluence of events this week that (A) the Nuffield Council, an ethics think tank, gives a thumbs up in a new report to heritable human genetic modification that would probably include using CRISPR in the same week that (B) a new paper reports that CRISPR can cause unpredictable genomic damage and several other concerning reports
Mixed Nuffield Council Report Too Aspirational on Human Genetic Modification Read More »
There’s much more to CRISPR-Cas9 than just gene editing and a new paper from the lab of Rudy Jaenisch in Cell highlights that in an exciting way. It reports epigenetic reversal of a Fragile X Syndrome phenotype in induced pluripotent stem cell (IPSC) neurons. Fragile X Syndrome is a neurological disorder in boys resulting from CGG repeat
Journal club: Jaenisch lab paper on epigenetic CRISPR-Cas9 rescue of Fragile X in a dish Read More »
Is President Donald Trump a fan of eugenics? He seems to believe that he comes from greatly superior genetic stock. He hasn’t said much of anything about CRISPR ‘gene editing’ technology that can readily alter the genetic code of cells of nearly any organism, including humans, but Trump has a fascination with the concept of
Wearing ‘Good Genes’: Trump & Eugenics Read More »
We can be confident that human genetic modification via CRISPR’ing of embryos soon will be safe and effective after that new exciting Mitalipov team paper, right? Wrong. The reality is far more complicated and interesting on the tech side. In a nutshell, I see the paper as a significant scientific, but not necessarily medical advance.
4 key reasons Mitalipov paper doesn’t herald safe CRISPR human genetic modification Read More »
Are designer babies made using CRISPR or other genetic modification technologies closer to reality today? If so, what exactly should we do about it? Researchers can use CRISPR to genetically modify just about any organism or its cells, but targeting humans is the subject of the most intense discussion including using CRISPR in the human germline
CRISPR, human genetic modification, & a needed course correction Read More »