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Gene Editing

All articles tagged with #gene editing

Embryo base editing nudges germline research toward reality, intensifying designer-baby debate
science3 days ago

Embryo base editing nudges germline research toward reality, intensifying designer-baby debate

New studies using base editing in early human embryos show precision editing with fewer chromosomal errors but still face mosaicism and off-target effects. While these results advance understanding of embryo development and disease-related genes, experts warn that safety, ethical, and regulatory hurdles remain, keeping the door to germline editing and “designer babies” firmly closed for now rather than opened.

via CNN|
#base-editing#crispr#embryos
CRISPR’s Next Act: From Bacterial Defense to One-Time Therapies and Global Ethics
science1 month ago

CRISPR’s Next Act: From Bacterial Defense to One-Time Therapies and Global Ethics

Jennifer Doudna describes how CRISPR-Cas9 evolved from a bacterial immune system into a programmable gene-editing tool, its rapid uptake across medicine and agriculture, and the ongoing delivery challenges to different tissues. The discussion covers a landmark 2024 liver-targeted therapy for Baby KJ, the prospect of one-time genetic treatments, and the ethical, regulatory, and accessibility questions raised by germline edits (notably the 2018 case), underscoring the need for cautious public engagement as the CRISPR toolbox expands.

via Quanta Magazine|
#crispr#delivery#ethics
Investment-Driven Breakthroughs Put Cancer and Chronic Disease in Focus
health1 month ago

Investment-Driven Breakthroughs Put Cancer and Chronic Disease in Focus

Decades of biomedical investment are yielding notable advances, including a pancreatic cancer therapy that doubles survival in late-stage trials, an obesity drug with powerful weight loss, and early Gene-editing approaches that could lower disease risk; while these advances extend life and hint at cures, high costs and access barriers persist, underscoring the need for sustained funding and a stronger emphasis on prevention.

via Axios|
#biotech#cancer#gene-editing
NIH-backed STEP brain-delivery tech raises questions about brain gene editing
science1 month ago

NIH-backed STEP brain-delivery tech raises questions about brain gene editing

Yale researchers secured a $40M NIH grant to advance STEP, a 10-nm brain-delivery platform for CRISPR gene editors; early preclinical work in mice hints at brain-wide delivery and functional gains, but the mechanism remains unclear, safety and scaling concerns persist, and full details won’t be published until 2028 as clinical work progresses.

via The Transmitter|
#angelman-syndrome#brain-delivery#gene-editing
Biotech Barbie bets on openly editing embryos to prevent disease
technology1 month ago

Biotech Barbie bets on openly editing embryos to prevent disease

Canadian entrepreneur Cathy Tie, nicknamed the “Biotech Barbie,” is pursuing openly regulated germline gene editing to prevent inherited diseases through Origin Genomics, after a controversial past with He Jiankui and a string of biotech ventures. She argues for regulator-approved, transparent research and says the field’s progress is inevitable, while critics warn about safety and the ethical implications for future generations. The piece frames her effort against a backdrop of private funding, a race among biotech startups, and the broader debate over embryo editing versus embryo selection.

via The Guardian|
#bioethics#embryo-editing#gene-editing
One-shot gene-editing therapy reduces LDL by up to 62% in early trial
science1 month ago

One-shot gene-editing therapy reduces LDL by up to 62% in early trial

In a small Phase I study, Verve Therapeutics’ VERVE-102, a one-time mRNA-based gene-editing therapy targeting PCSK9, reduced LDL by up to 62% in the highest-dose group among 35 participants, with no serious safety events aside from a transient mild rise in a liver enzyme; results suggest durable LDL-C lowering, but larger and longer trials are needed for confirmation, and the FDA has granted Fast Track designation.

via Ars Technica|
#gene-editing#ldl#pcsk9
Intellia's in vivo CRISPR therapy hits Phase 3 for hereditary angioedema, signaling a potential one-time treatment
science2 months ago

Intellia's in vivo CRISPR therapy hits Phase 3 for hereditary angioedema, signaling a potential one-time treatment

Intellia Therapeutics said its one-time, in vivo CRISPR-based treatment for hereditary angioedema met its Phase 3 primary endpoint, reducing attacks by 87% vs placebo and achieving 62% attack-free patients at six months after a liver-directed infusion, with a favorable safety profile. The company has started a rolling FDA submission and targets a U.S. launch in 2027 if approved.

via CNBC|
#crispr#gene-editing#hereditary-angioedema
Boston scientists win big at Breakthrough Prizes, dubbed science's Oscars
biotech2 months ago

Boston scientists win big at Breakthrough Prizes, dubbed science's Oscars

Four Boston-area researchers were honored at the 2026 Breakthrough Prize ceremonies: Stuart Orkin and Lee Roberts received Breakthrough Prizes of $3 million each for gene editing and muon physics, while Dillon Brout (Boston University) and Shu-Heng Shao (MIT) were New Horizon Prize recipients, each $100,000, for cosmology and generalized symmetries in quantum field theory. The awards, called the 'Oscars of Science' and cofounded by Zuckerberg and Chan, celebrate long-term breakthroughs—from sickle cell gene-editing therapies to precision cosmology data and new physics ideas.

via The Boston Globe|
#biotech#boston-science#breakthrough-prize
Tiny CRISPR enzyme boosts in-body gene editing for targeted therapies
science2 months ago

Tiny CRISPR enzyme boosts in-body gene editing for targeted therapies

NIH-backed researchers identified the small Al3Cas12f CRISPR enzyme and engineered the Al3Cas12f RKK variant to dramatically improve editing in human cells, raising efficiency from under 10% to over 80% (up to 90% in a key region). This enzyme's size allows packaging into AAV delivery vectors, a major step toward in-body gene therapies for diseases like cancer and ALS; next, researchers will test AAV-packaged delivery to move toward clinical use.

via National Institutes of Health (.gov)|
#aav#cas12f#crispr
Colossal’s de-extinction bid stirs science and ethics debate
science3 months ago

Colossal’s de-extinction bid stirs science and ethics debate

Dallas-based Colossal Biosciences is pursuing revival of extinct species—including dire wolves and the dodo—using ancient DNA and CRISPR. The effort has drawn praise for pushing conservation tech and public engagement, but also sharp criticism from scientists who question feasibility and ecological risks, warn against conflating revived animals with extinct ones, and worry about undermining habitat protection; supporters see potential conservation benefits and genetic diversity applications, while critics urge caution and rigorous science.

via The Guardian|
#colossal-biosciences#conservation#crispr
Gene-edited fungus could turbocharge meat substitutes and slash environmental footprint
technology4 months ago

Gene-edited fungus could turbocharge meat substitutes and slash environmental footprint

A gene-edited fungus used to make mycoprotein grows protein nearly twice as fast with far less sugar after deleting two genes with CRISPR to thin its cell wall; lifecycle analyses across six countries show substantial reductions in climate emissions, land use, and freshwater pollution, plus improved essential amino-acid quality. Regulators and large-scale production hurdles remain before it reaches diners.

via Earth.com|
#crispr#fungus#gene-editing