An NIH-supported team has delivered the first personalized CRISPR-based gene therapy to an infant with CPS1 deficiency, offering new hope for rare disease treatment. The customized approach shows early success and may pave the way for future individualized therapies...
MIT engineers have developed a gene circuit that precisely controls gene expression, offering potential breakthroughs in gene therapy for disorders like fragile X syndrome. This advancement helps ensure therapeutic genes are expressed at optimal levels, minimizing risks and paving the way for more effective treatments...
A new preclinical study shows that Imiquimod enhances immune responses to an HIV DNA vaccine, offering promising implications for nucleic acid vaccine design and innate immune modulation strategies...
A new study offers hope for improving CAR-T cell therapy in treating B-cell Acute Lymphoblastic Leukemia (B-ALL), a cancer with high relapse rates. Researchers have developed an innovative approach involving a TIM-3 decoy that prevents immune evasion by leukemia cells...
TIGR (Tandem Interspaced Guide RNA) systems use RNA guides to target specific DNA sites, offering a compact and modular alternative to CRISPR. Scientists at MIT and the Broad Institute discovered over 20,000 TIGR-associated proteins, some capable of precise DNA cutting, potentially expanding genome...
A groundbreaking gene therapy using rAAV8.hRKp.AIPL1 has restored vision in 11 children with AIPL1-related retinal dystrophy (LCA4), who were blind at birth. Published in The Lancet, the study shows significant improvements in visual acuity and retinal preservation, supporting expedited approval...
Patients in England with severe beta-thalassaemia will be amongst the first in Europe to benefit from one-time gene therapy exagamglogene autotemcel. Exa-cel is the world’s first CRISPR-based gene therapy and the first gene therapy available in Europe for treating severe beta-thalassaemia...
Two hallmark toxicities of chimeric antigen receptor (CAR) T-cell therapy may be rare after 2 weeks following infusion in patients with diffuse large B-cell lymphoma (DLBCL), supporting a shorter, more flexible toxicity monitoring period, according to a recent study...
CAR T-cell therapies show promise for blood cancers but struggle with solid tumors due to T-cell weakness in tumor microenvironments. Researchers find genetic alterations in cancerous T cells, giving engineered T cells "superpowers" to effectively kill tumors without behaving like cancer cells...
In a groundbreaking development that could revolutionize organ transplantation, eGenesis, a biotechnology company dedicated to addressing the global organ shortage crisis, has announced the successful transplantation of a genetically engineered porcine kidney into a living human recipient...
