Published on: 2025-05-20 05:23:07
A team of doctors and scientists have successfully treated a rare genetic condition with the first-ever personalized gene-editing therapy. Results of the groundbreaking treatment have been published in The New England Journal of Medicine, with an accompanying editorial by a doctor who had previously overseen the FDA's gene-therapy regulation efforts. The patient in this historic case was KJ, an infant born with CPS1 deficiency, which has about a 50 percent mortality rate within the first week.
Keywords: crispr editing gene human kj
Find related items on AmazonPublished on: 2025-05-20 17:00:00
Last August, KJ Muldoon was born with a potentially fatal genetic disorder. Just six months later, he received a Crispr treatment designed just for him. Muldoon has a rare disorder known as CPS1 deficiency, which causes a dangerous amount of ammonia to build up in the blood. About half of babies born with it will die early in life. Current treatment options—a highly restrictive diet and liver transplantation—aren’t ideal. But a team at the Children’s Hospital of Philadelphia and Penn Medicine w
Keywords: ammonia crispr gene kj treatment
Find related items on AmazonPublished on: 2025-05-25 14:10:00
The CRISPR patents are back in play. Yesterday, the US Court of Appeals for the Federal Circuit said scientists Jennifer Doudna and Emmanuelle Charpentier will get another chance to show they ought to own the key patents on what many consider the defining biotechnology invention of the 21st century. The pair shared a 2020 Nobel Prize for developing the gene-editing system, which is already being used to treat various disorders. But when US patent rights were granted in 2014 to Feng Zhang of
Keywords: crispr doudna patents read story
Find related items on AmazonPublished on: 2025-05-31 00:33:11
CRISPRa browning of human white adipocytes To induce browning in human adipocytes, we used CRISPRa to upregulate UCP1, PPARGC1A or PRDM16, all known genes involved in BAT development and function. Using CRISPick36, we designed five guide RNAs (gRNAs) targeting each gene’s promoter and cloned them into an adeno-associated virus (AAV)-based expression vector. Differentiated adipocytes derived from human white preadipocytes were co-transfected with the gRNAs along with a Staphylococcus aureus endo
Keywords: adipocytes crispra fig organoids ucp1
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