@danielevanbauer.bsky.social
@sebastien-levesque.bsky.social and I review therapeutic genome editing for inherited blood disorders rdcu.be/ewcZ3. We cover CRISPR, patient journey, DNA repair, patient access, genotoxicity, hemoglobinopathies, delivery, bone marrow failure, coselection, immunodeficiency, and more.
CRISPR-based therapeutic genome editing for inherited blood disorders
Nature Reviews Drug Discovery - CRISPR-based technologies provide a diverse set of tools to correct pathogenic mutations. This Review describes the promise and the challenges of genome editing...
rdcu.be
July 15, 2025 at 12:44 AM
@sebastien-levesque.bsky.social and I review therapeutic genome editing for inherited blood disorders rdcu.be/ewcZ3. We cover CRISPR, patient journey, DNA repair, patient access, genotoxicity, hemoglobinopathies, delivery, bone marrow failure, coselection, immunodeficiency, and more.
Excited to share our latest where we describe a gene editing method termed prime assembly that enables RNA programmable targeted integration of gene-sized DNA sequences in nondividing cells without dsDNA or nuclease-driven DSBs. See thread by incomparable @Seb. Also complementary work Sontheimer/Xue
I’m thrilled to share our latest preprint! Inspired by isothermal Gibson assembly cloning, we wondered whether we could assemble and integrate DNA sequences at a specific genomic locus in human cells. A thread (1/7) www.biorxiv.org/content/10.1...
www.biorxiv.org
June 18, 2025 at 9:46 PM
Excited to share our latest where we describe a gene editing method termed prime assembly that enables RNA programmable targeted integration of gene-sized DNA sequences in nondividing cells without dsDNA or nuclease-driven DSBs. See thread by incomparable @Seb. Also complementary work Sontheimer/Xue
Reposted
In @nature.com we describe the use of scalable #proteinengineering & #machinelearning to predict millions of bespoke CRISPR enzymes, offering safer & more efficient genome editing tools 🧬🖥️ @rachelsilverstein9.bsky.social @mgbresearch.bsky.social @harvardmed.bsky.social
www.nature.com/articles/s41...
www.nature.com/articles/s41...
Custom CRISPR—Cas9 PAM variants via scalable engineering and machine learning - Nature
Nature - Custom CRISPR—Cas9 PAM variants via scalable engineering and machine learning
www.nature.com
April 22, 2025 at 8:57 PM
In @nature.com we describe the use of scalable #proteinengineering & #machinelearning to predict millions of bespoke CRISPR enzymes, offering safer & more efficient genome editing tools 🧬🖥️ @rachelsilverstein9.bsky.social @mgbresearch.bsky.social @harvardmed.bsky.social
www.nature.com/articles/s41...
www.nature.com/articles/s41...
Reposted
New paper from my lab! Elevated mitochondrial membrane potential is a therapeutic vulnerability in Dnmt3a-mutant clonal hematopoiesis: rdcu.be/eh1Nb Complementary to new work from Steven Chan & @georgevassiliou.bsky.social as described by @goodell-lab.bsky.social: www.nature.com/articles/d41...
Age-related blood condition counteracted with a common diabetes drug
Metformin could reduce people’s risk of certain age-associated blood cancers, but more work is needed to identify who is likely to benefit.
www.nature.com
April 16, 2025 at 9:02 PM
New paper from my lab! Elevated mitochondrial membrane potential is a therapeutic vulnerability in Dnmt3a-mutant clonal hematopoiesis: rdcu.be/eh1Nb Complementary to new work from Steven Chan & @georgevassiliou.bsky.social as described by @goodell-lab.bsky.social: www.nature.com/articles/d41...
Reposted
Francis Collins, the NIH Director for 12 years, led the Human Genome Project and other NIH efforts for 32 years, resigned today. Key words from his resignation letter
www.nytimes.com/2025/03/01/u...
www.nytimes.com/2025/03/01/u...
March 1, 2025 at 6:07 PM
Francis Collins, the NIH Director for 12 years, led the Human Genome Project and other NIH efforts for 32 years, resigned today. Key words from his resignation letter
www.nytimes.com/2025/03/01/u...
www.nytimes.com/2025/03/01/u...