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UCLA advances CRISPR gene-editing therapy for sickle cell disease

A UCLA-led team has described how they advanced an experimental CRISPR approach for sickle cell disease into a blood stem cell therapy that can be manufactured at the scale and quality required for a clinical trial.

4 Nov 2026

Ancient virus may help explain differences in human and chimpanzee brains

Pandemics can arise wherever animal or human populations are sufficiently large, and they are not confined to the modern age.

23 Oct 2026

Stem cell therapies may need new protein quality control standards

Current Good Manufacturing Practice (GMP) standards for cell therapies assess identity, viability, sterility, and genetic stability. For extracellular vesicle (EV) products, Minimal Information for Studies of Extracellular Vesicles (MISEV2023) guidance adds particle size, concentration, markers, morphology, contaminants, endotoxin, and assays.

22 Oct 2026

Long non-coding RNAs offer new pathways to overcome cancer treatment resistance

Scientists are highlighting long non-coding RNAs (lncRNAs) as promising targets in the effort to overcome one of cancer treatment's most persistent challenges: therapeutic resistance.

12 Oct 2026

Streamlining CRISPR workflows, from editing to analysis

INTEGRA's automated solutions optimize CRISPR workflows, enhancing gene editing efficiency and accelerating the path to innovative therapeutic developments.

From INTEGRA Biosciences 30 Sep 2026

Integrated DNA Technologies and Aldevron launch new Cas9 mRNA solutions to accelerate path from gene editing research to development

Danaher companies Integrated DNA Technologies (IDT), a global leader in genomics, and Aldevron, a premier manufacturing partner for DNA, RNA, and protein, today announced the launch of research grade S.p. Cas9 mRNA in wild type and SpyFi™ high-fidelity formats.

From Integrated DNA Technologies 18 Sep 2026

Scientists discover tiny protein region that shapes tooth enamel

Tooth enamel looks simple from the outside and consists of a hard, smooth surface built to withstand years of wear.

11 Sep 2026

Tumor environments sculpt diverse natural killer cells for cancer therapies

For decades, natural killer (NK) cells have been classified into two main groups based on surface markers: one specialized for cytokine production and the other for direct killing.

4 Sep 2026

Generative AI helps scientists design entirely new RNA transporters

Researchers at Helmholtz Munich and TUM have developed Synthetic Transfer Vehicles (STVs), an entirely new class of RNA transporters.

2 Sep 2026

Gene editing fails to protect beta-cell transplants from rejection

In people with type 1 diabetes, the immune system mistakenly destroys the pancreatic beta cells responsible for producing insulin. Patients therefore require lifelong insulin therapy to regulate their blood glucose levels.

19 Aug 2026

Researchers identify genes that hinder CRISPR editing efficiency

Like a delivery driver navigating crowded city streets, a gene-therapy-toting lipid nanoparticle faces a gauntlet of potential detours on its journey toward a cell's nucleus.

13 Aug 2026

Genetic analysis of 1 million people points to FNIP1 as a promising metabolic target

Mega-scale exome sequencing of 1,032,116 people identified 59 independent genes linked to the TG:HDL ratio, including 44 rare-coding associations not detected in the previous largest analysis. Ultra-rare FNIP1 loss-of-function variants were associated with favorable fat distribution, lower liver fat and glycemia, and around 60% lower odds of a composite cardiometabolic disease outcome.

7 Aug 2026

New study reveals genetic drivers of inherited bone marrow failure syndrome

Inherited bone marrow failure syndrome, or IBMFS, describes a group of disorders in which inherited genetic abnormalities impair the bone marrow's ability to produce sufficient healthy blood cells.

6 Aug 2026

Advances in DNA technology are reshaping preventive healthcare

A new review highlights how advances in DNA technology are reshaping the future of healthcare by shifting the focus from treatment to prevention.

6 Aug 2026

Long-acting HIV prevention tools could help close the gap before a vaccine arrives

A review of non-vaccine HIV interventions highlights the strong preventive effects of long-acting PrEP, sustained ART, U=U, vertical transmission programs, and voluntary medical male circumcision. Gene editing and multispecific antibodies may expand future options, but safety, cost, access, adherence, and implementation barriers remain.

5 Aug 2026

Advances in X chromosome inactivation open therapeutic opportunities for X-linked genetic disorders

A new review explores how advances in understanding X chromosome inactivation (XCI) are creating potential therapeutic opportunities for a range of X-linked genetic disorders, including Rett syndrome, Fabry disease, Duchenne muscular dystrophy, hemophilia, and others.

3 Aug 2026

New OpenABE gene editor improves editing efficiency by up to 36 times

A joint research team led by Professor Daesik Kim (Department of Medicine, Sungkyunkwan University), along with Professor Yong-Sub Kim (University of Ulsan College of Medicine) and Professor Jae-Hyun Park (Sungkyunkwan University School of Medicine), has announced the development of 'OpenABE' (Open Adenine Base Editor).

30 Jul 2026