An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...

A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...

Rare diseases, defined by FDA as “a disease or condition that affects less than 200,000 people in the United States” (1), may ...

Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA ...

A new CRISPR approach can control genes without cutting DNA, opening a safer path for treating genetic diseases. A newly ...

Menlo Ventures has made a $16 million bet that the “ baby KJ ” custom CRISPR therapy success story is repeatable. The funding ...

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...

Abu Dhabi successfully completes its first gene therapy for genetic blood disorders, revolutionizing treatment approaches for ...

Expected rapid advances in CAR T and other cellular therapies may expand treatment options not only in cancer but also in ...

Gene therapy advancements necessitate improved delivery infrastructure and comprehensive data collection across age groups to address logistical complexities and policy gaps. Multidisciplinary ...

Ascidian Therapeutics ("Ascidian"), a biotechnology company seeking to treat human diseases by rewriting RNA, and Forge ...

Cell and gene therapy are revolutionary approaches to treating diseases at the molecular level. They involve the manipulation ...