CRISPR-Mediated Correction of ABCA4 Mutations in Stem Cells May Provide Treatment for Incurable Inherited Disease Causing Vision Loss

CRISPR-Mediated Correction of ABCA4 Mutations in Stem Cells May Provide Treatment for Incurable Inherited Disease Causing Vision Loss - Blog

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 […]

Delivering the next generation of cancer immunotherapies with RNA – Cell 

Delivering the next generation of cancer immunotherapies with RNA - Cell  - Blog

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

UW researchers engineer stem cells that do not generate dangerous arrhythmias - Blog

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

Appetite for Destruction: The Indiscriminate Nuclease Activity of Cas12a2 - Blog

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 […]

Liquid biopsy predicts response to CAR-T for multiple myeloma

Liquid biopsy predicts response to CAR-T for multiple myeloma - Blog

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

Bispecific LV20.19 CAR T Cells Induce 92% CR Rate in Relapsed/Refractory Mantle Cell Lymphoma - Blog

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 […]