UCSF researchers hope to cure sickle cell disease through CRISPR gene editing
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
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
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 […]
Rapid manufacturing of non-activated potent CAR T cells | Nature Biomedical Engineering
In the process of engineering CAR T cells, if the patient’s T cells are out of the body for too long, they can lose their ability to replicate, which is critical to their effectiveness as a live drug. Thus, a research team has developed a new approach that reduces the time needed to modify patients’ […]
Bioinstructive implantable scaffolds for rapid in vivo manufacture and release of CAR-T cells | Nature Biotechnology
Many people are excluded from CAR T cell-based treatments because of its cost. One reason for the high cost is that the manufacturing process is complex, time-consuming and must be individually tailored to each cancer patient. So to address this challenge, the researchers created a biotechnology called Multifunctional Alginate Scaffolds for T cell Engineering and […]
Researchers develop new precision medicine technology for cancer immunotherapy
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 […]
Nonviral CAR-T is ‘remarkably safe’ and clinically active in B-cell ALL
CARCIK-CD19, an investigational therapy developed by scientists in Italy, involves donor-derived CD19-targeted CAR T cells differentiated according to the cytokine-induced killer (CIK) cell protocol that uses non-viral Sleeping Beauty transposon gene transfer to prevent graft-versus-host disease. More than 60% of patients with relapsed or refractory B-cell acute lymphoblastic leukemia achieved initial complete remission after receiving […]
Clonal relations in the mouse brain revealed by single-cell and spatial transcriptomics | Nature Neuroscience
The mammalian brain contains many specialized cells that develop from a thin sheet of neuroepithelial progenitor cells. However, the lineage relationships between mature cell types and progenitor cells are not well understood. Therefore, researchers performed high-throughput clonal tracking and expression profiling of mouse forebrain cells using single-cell and spatial transcriptomics and found two populations of […]
CRISPR activation and interference screens decode stimulation responses in primary human T cells – Science
CRISPR genome editing has served as a powerful tool to delete or modify DNA sequences and study the resulting effect. Now, researchers at the Gladstone Institutes and UC San Francisco (UCSF) have co-opted the CRISPR-Cas9 system to forcibly turn on genes rather than edit them in human immune cells. The method, known as CRISPRa, allowed […]
Case study highlights Parkinson’s-like toxicity after BCMA-directed CAR T-cell therapy
Clinicians treating patients with CAR T cells have become adept at identifying and treating acute neurotoxicity, a common adverse event associated with the therapy. Researchers at Mount Sinai published a case study in Nature Medicine of a patient who developed a neurocognitive, hypokinetic movement disorder with features of Parkinson’s disease after receiving the CAR-T therapy […]