CRISPR-Mediated Correction of ABCA4 Mutations in Stem Cells May Provide Treatment for Incurable Inherited Disease Causing Vision Loss
Stargardt’s disease is an inherited macular neurodegenerative disorder that causes retinal dystrophy and vision loss. Stargardt disease is caused by mutations in the ABCA4 gene, which encodes a protein involved in the visual cycle and the transport of toxic photoproducts out of the retina. Currently, there is no treatment for Stargardt’s disease, and although restoring […]
ST3GAL1 and βII-spectrin pathways control CAR T cell migration to target tumors | Nature Immunology
Immunotherapy, particularly CAR T-Cell cancer therapy, extends the lives of many patients. But sometimes the therapy randomly migrates to places it shouldn’t go, sneaking into the lungs or other non-cancerous tissue and causing toxic side effects. However, a team of researchers has discovered the molecule responsible for guiding T cells to tumors, setting the stage […]
Delivering the next generation of cancer immunotherapies with RNA – Cell
Immunotherapy is an essential component of cancer treatment. However, despite the success of immunotherapies for various types of cancer, only a few cancer patients have shown responses to current immune therapies and therapies can have adverse effects. Therefore, researchers have created intramuscular messenger ribonucleic acid (mRNA) lipid nanoparticle (LNP) vaccines as likely candidates for therapeutic […]
UW researchers engineer stem cells that do not generate dangerous arrhythmias
Researchers have engineered stem cells that do not generate dangerous arrhythmias, a complication that has until now thwarted efforts to develop stem cell therapies for injured hearts. Researchers suspected that transplanted stem cells that did not generate arrhythmias were behaving like early embryonic cells chaotically generating signals and causing dangerous heart rhythms. So the researchers […]
Appetite for Destruction: The Indiscriminate Nuclease Activity of Cas12a2
Despite its phylogenetic placement with DNA-targeting nucleases, Cas12a2 targets and cleaves RNA. When researchers put Cas12a2 in test tubes with pure DNA and a guide, nothing happened, because its target was not present and it remained inactive. However, sometimes a small amount of activating RNA was present, contaminating the sample. When the RNA was present […]
Real-World Analysis Evaluates Inpatient Vs Outpatient CAR T-Cell Therapy Administration in MCL and F
As CAR T-cell therapies have gained approvals in relapsed/refractory mantle cell lymphoma (MCL) and follicular lymphoma (FL) patient populations, there is continued interest in the optimal administration of this treatment option. CAR T-cell therapy is typically administered in the hospital setting, but as the therapy continues to become more widely used in clinical practice, there […]
Study focuses on new approach that delivers a “one-two punch” to help T cells attack solid tumors
One of the challenges of CAR T cell therapy in solid tumors is a phenomenon known as T cell exhaustion. Previous studies have alluded to the inflammatory regulator Regnase-1 as a potential target to indirectly overcome the effects of T-cell exhaustion, as it can cause hyperinflammation when disrupted in T cells, reviving them to produce […]
Engineered human hepatocyte organoids enable CRISPR-based target discovery and drug screening for steatosis | Nature Biotechnology
The accumulation of fat in the liver is an increasingly common disease worldwide, with more than a quarter of the world’s population affected. Various causes can lead to the development of fatty liver, with diet and lifestyle being the most common contributors. There is currently no treatment for fatty liver that can stop or reverse […]
Liquid biopsy predicts response to CAR-T for multiple myeloma
Multiple myeloma is difficult to treat because of the heterogeneity of genomic aberrations and responses to treatment. Currently, bone marrow examinations are used for genomic evaluation at diagnosis, at evaluation of minimal residual disease and at relapse. This is an invasive procedure, but it also carries the risk of false negatives due to spatial heterogeneity. […]
Bispecific LV20.19 CAR T Cells Induce 92% CR Rate in Relapsed/Refractory Mantle Cell Lymphoma
Mantle cell lymphoma (MCL) is an aggressive B-cell malignancy that researchers believe is not curable using current standard approaches. However, researchers developed a bispecific CAR T-cell therapy targeting both CD20 and CD19 cells induced a 92% complete response (CR) rate at 90 days in patients with relapsed/refractory MCL. In the study, researchers administered CAR T […]
