All articles tagged with #gene editing
A weekend reads roundup from Retraction Watch highlights a second death in a Chinese gene-therapy trial, the resignation of Cambridge professor Jason Arday amid plagiarism allegations, and Retraction Watch’s 16th birthday, alongside extensive coverage of data integrity, paper mills, AI-related misconduct, and global debates about trust in scholarly publishing.
Researchers used CRISPR to disable the gene that drives allergy-causing proteins in dog saliva and fur, creating two hypoallergenic beagles, Bailey and Alfie. Early tests showed no allergy protein in their saliva or hair, and a skin-prick test on a human volunteer suggested reduced reaction. While the method is a first for dogs, experts warn it will be years before any pet becomes available, and long-term safety, cost, and ethical implications—such as potentially dampening adoption of shelter animals—must be addressed before commercialization.
Kindred Companion Sciences used CRISPR to knock out the Can f 1 allergen gene in beagles, producing Bailey and Alfie. Edited dogs showed no detectable Can f 1 in saliva or dander, and a skin-prick test on a volunteer who lived with Bailey produced no reaction (non-edited dog samples did). The team used a cloning-like method (somatic cell nuclear transfer) to create the embryos and plans to expand to other breeds and service dogs, though a public timeline and broader safety checks remain ongoing.
Nature is reviewing concerns about a February paper from Shanghai Jiao Tong University describing preclinical gene-editing work for SNIBCPS after a 6-year-old girl died from an immune reaction to the therapy; the death and the family’s funding were not disclosed in the manuscript, prompting an Editors’ Note and an investigation by the university. The case has drawn international media attention and ongoing scrutiny of the study’s data and disclosures.
A young girl died in an experimental brain-targeted gene-editing trial at Shanghai Jiaotong University, prompting an ethics investigation and highlighting gaps in oversight amid China's booming biotech sector; authorities have since tightened rules around investigator-initiated trials and reporting of adverse events, but observers warn that more safeguards are needed to prevent recurring ethical lapses.
A Chinese family paid about $860,000 for an experimental brain-targeted base-editing therapy for their 6-year-old daughter with a developmental disorder. The treatment, never used in humans, preceded fatal complications—thrombotic microangiopathy—days after administration, prompting scrutiny of safety signals from animal studies, questions about research oversight, and ethics concerns after a Nature paper published without acknowledging the child's case.
An investigation has been launched into neuroscientist Zilong Qiu after a six-year-old girl died following an experimental base-editing gene therapy, funded by the family with over $800,000. Qiu allegedly did not publicly disclose the death and altered a related Nature paper to omit funding details and human-trial references. The trial, which used CRISPR-based edits delivered via trillions of viruses to Mei’s brain, is believed to have caused a fatal immune reaction; the hospital was fined a small amount for oversight. Outside experts urge Nature to review the work, and the case echoes China’s past genome-editing scandals including He Jiankui.
A six-year-old girl in China died a week after receiving an experimental spinal infusion of viruses designed to reach the brain and correct a rare non-life-threatening genetic condition, in a trial funded by her parents who paid more than $800,000. Investigators say the risks were downplayed and that researchers proceeded with low odds of success, prompting calls for a full data review and raising questions about testing such therapies in non-fatal conditions and the path from preclinical work to clinical use.
FDA-approved enlicitide is an oral pill that lowers LDL cholesterol by about 60%, offering a needle-free alternative to injections and statins for those who can’t tolerate them; the drug’s development builds on PCSK9 genetics, and while LDL reductions look promising, hard outcomes like deaths will take years to prove, and cost and access—alongside the potential of gene editing and other therapies—will shape how broadly it helps curb heart disease.
An exclusive investigation shows a one-patient Shanghai trial using a brain-targeted base editor ended in the death of a 6-year-old girl from a severe immune reaction; her family paid more than $800,000 to fund the therapy, and the trial’s funding and patient details were not publicly disclosed, even as the related Nature paper glossed over them. Experts call for a full data review and potential retraction amid concerns about oversight and safety.
Boston Children’s Hospital, the Broad Institute, and Jackson Laboratory are launching the Center for Therapeutic Genetics, a nonprofit collaboration to develop and share programmable gene-editing therapies for rare diseases and make them more scalable and accessible by building reusable platforms—models, manufacturing data, safety data, and clinical programs—so doctors can deliver these treatments like standard medical procedures; backed by ARPA-H with up to $160 million, the center will initially target pediatric genetic epilepsies and is in its founding phase, not yet accepting patient referrals.
Researchers used AI to design synthetic CRISPR nucleases not seen in nature, aiming to edit genes more efficiently. By reverse-engineering DNA templates to preserve the proteins’ final shape, the team generated thousands of candidate enzymes from TnpB precursors, suggesting a faster path to novel genome-editing tools with potential applications in medicine and agriculture.
Nobel laureate Jennifer Doudna and colleagues report an AI-driven platform for designing non-natural nucleases, producing a tiny TnpB enzyme capable of binding and cutting DNA in human, plant, and bacterial cells. By using an inverse protein-model to tailor sequences to predefined backbones, the team envisions on-demand enzyme design with potential medical and agricultural applications and notes possible IP implications as the field advances.
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.
Scribe Therapeutics filed for a Nasdaq IPO to gauge investor appetite for early-stage biotech and gene-editing startups, backed by major investors including RA Capital, Andreessen Horowitz, OrbiMed and Avoro Capital.