PD-1 Blockade in Mismatch Repair–Deficient, Locally Advanced Rectal Cancer | NEJM

PD-1 Blockade in Mismatch Repair–Deficient, Locally Advanced Rectal Cancer | NEJM - Blog

Breakthrough findings were presented at the 2022 ASCO Annual Meeting and published in The New England Journal of Medicine by researchers at Memorial Sloan Kettering Cancer Center (MSK) confirming a clinical complete response in all 14 patients who received the immunotherapy treatment dostarlimab as a first-line treatment for mismatch repair-deficient (MMRd) locally advanced rectal cancer. This new approach of “immunoablative” […]

CRISPR/Cas9 deletions induce adverse on-target genomic effects

CRISPR/Cas9 deletions induce adverse on-target genomic effects - Blog

In a recent study, a team of researchers used a CRISPR/Cas9 system to evaluate the use of tRNA by deleting two tRNA genes from the genomes of hyperhepatocellular carcinoma and human chronic myeloid quasi-haploid leukaemia cells. The authors discovered numerous unexpected genomic changes at the target region using an improved droplet-based target enrichment approach followed […]

UCSF researchers hope to cure sickle cell disease through CRISPR gene editing

UCSF researchers hope to cure sickle cell disease through CRISPR gene editing - Blog

With a $17 million grant, UCSF Benioff Children’s Hospital in Oakland will launch a four-year trial evaluating CRISPR/Cas9 gene editing as a potential cure for sickle cell disease. The trial is the first to evaluate the use of non-viral CRISPR gene editing tools to correct a harmful mutation in the beta-globin gene in a patient’s […]

FDA grants fast track designation to CD33-directed CAR-T for acute myeloid leukemia

FDA grants fast track designation to CD33-directed CAR-T for acute myeloid leukemia - Blog

The FDA has granted accelerated designation to PRGN-3006 for the treatment of adults with relapsed or refractory acute myeloid leukemia. PRGN-3006 (Precigen Inc.) is a CAR T cell therapy that targets the CD33 protein on the surface of cancer cells. This investigational therapy, designed to reduce manufacturing time to 1 day, is based on Precigen’s […]

 Nasal Spray Sends CRISPR to the Brain

 Nasal Spray Sends CRISPR to the Brain - Blog

Mucopolysaccharidosis I is caused by mutations in the gene for the enzyme alpha-L-iduronidase (IDUA). This enzyme is required for the breakdown of substances called glycosaminoglycans (GAGs) that are by-products of chemical reactions in the body’s cells. In 2018, a team of researchers was able to demonstrate the feasibility of CRISPR gene editing in vivo in […]

Researchers develop new precision medicine technology for cancer immunotherapy

Researchers develop new precision medicine technology for cancer immunotherapy - Blog

CAR T-cell therapy and antibody treatments are two types of targeted immunotherapies that have revolutionized the fields of cancer care. However, there are still significant challenges in identifying cancer cell surface proteins as targets for immunotherapies. A research group at Lund University in Sweden is well on their way as they have developed a new […]