Today "a milestone in the evolution of personalized therapies for rare & ultra-rare inborn errors of metabolism"
—the 1st human to undergo custom genome editing
—outgrowth of decades of NIH funded research
https://t.co/HmJxYWDCV7
https://t.co/4DsNELnBut @NEJM
https://t.co/y7NMGPpXnE
@KarthikIndrAnna India has 1.4 billion people. If a few leave and succeed abroad, India won’t sink. Let’s focus on building a nation they’ll want to return to.
"Highly efficient #CRISPR/Cas9-mediated exon skipping for recessive dystrophic epidermolysis bullosa"
du Rand A [..] Sheppard H.
Bioeng Transl Med. 2024-01-17.
https://t.co/UMWCqwMRm0
… detected large on-target structural variants (deletions up to >5 kb at a frequency of ~10%)
For those who believe that using base editors and prime editors is safe, it's essential to note that they employ nickases, causing a single-strand break and damage. Therefore, conducting thorough genotyping is imperative to select clones that are genetically suitable.
"Detailed mechanisms for unintended large DNA deletions with #CRISPR, base editors, and prime editors"
Hwang GH, Lee SH [..] Bae S
bioRxiv 2024-01-05 (preprint)
https://t.co/T3k7dDydz3
CRISPRi screen with Nanopore seq. >
TMEJ is the dominant pathway generating large deletions.
"Detailed mechanisms for unintended large DNA deletions with #CRISPR, base editors, and prime editors"
Hwang GH, Lee SH [..] Bae S
bioRxiv 2024-01-05 (preprint)
https://t.co/T3k7dDydz3
CRISPRi screen with Nanopore seq. >
TMEJ is the dominant pathway generating large deletions.
"Effective prevention of atherosclerosis by non-viral delivery of #CRISPR/Cas9"
Xu C, Zhang X [..] Du J, Li Y, Xu F-J.
Nano Today. 2023-12-12.
https://t.co/6vFDOuNHSF
PuPGEA/pCas9-Pcsk9 composed of low-toxic and liver-targeting polycation
Today I am excited to share our preprint “Rapid DNA unwinding accelerates genome editing by engineered CRISPR-Cas9” from @doudna_lab, with co-authors @kaichen_1990, Kasia Soczek, @owen_t_tuck, @Ee_Doherty, @BW_Thornton, Bryant Xu, and Marena Trinidad. https://t.co/iIdHq255py
1/7
"Efficient gene editing in induced pluripotent stem cells enabled by an inducible adenine base editor with tunable expression"
Nandy K [..] Velayudhan SR. Sci Rep. 2023-12-11.
https://t.co/FtHtsxTOet
#CRISPR; AAVS1-iABE8e
“#CRISPR-Analytics (CRISPR-A): A platform for precise analytics and simulations for gene editing"
Sanvicente-García M [..] Güell M.
PLoS Comput Biol. 2023-05-30.
https://t.co/ocsQdfxCQs
CRISPR-A emerges as an evolution of CRISPR-GA, the first NGS-based method for ...
I'm revisiting Sapiens after 9 years, you’ll find that the book still holds up. The author beautifully narrates who we are, how we got here, and where we might be headed...
Sapiens: THE MULTI-MILLION COPY BESTSELLER https://t.co/xreRPsT6Bn
Precise CRISPR-Cas-mediated gene repair with minimal off-target and unintended on-target mutations in human hematopoietic stem cells https://t.co/aHGRuysmW0