Promising Results Shown For CRISPR
CRISPR has shown promising results in preliminary clinical studies to correct sickle cell disease and treat blindness, transthyretin amyloidosis, and hereditary angioedema. A new study in collaboration with University College London and Great Ormond Street Hospital for Children investigated the potential feasibility of base-edited CD7-targeted chimeric antigen receptor (BE-CAR7) T cells to induce remission in children with relapsed/refractory (r/r) CD7-positive T-cell acute lymphoblastic leukemia (T-ALL), prior to an allo-stem cell transplant (allo-SCT). To circumvent the challenges associated with human leukocyte antigen (HLA) barriers that prevent or severely hinder the use of CAR-T therapies, scientists have developed a CRISPR-based and guided cytidine deamination process that mediates a highly precise C→U→T conversion that directly disrupts gene expression without causing DNA breaks.
The novelty of the study included 4 key milestones. The first is deleting all existing T-cell cell surface markers with no need for HLA matching, making them universal and allowing for the possibility of having ready-made “off-the-shelf” CAR-T therapies. Second, remove CD7 to avoid T-cell fratricide when introduced to a new host. Third, the deletion of CD52, which makes T-cells invisible to some of the potent drugs the patient may receive during therapy. Finally, adding CAR that recognizes CD7 receptors, thus targeting T-cell leukemia. Using this technology, a 13-year-old girl named Alyssa was the first ever reported to receive the BE-CAR7 therapy. Diagnosed in 2021 and after chemotherapy and a bone marrow transplant, before the based-edited treatment, her medical team could not control her cancer. She entered remission within a month after the administration of BE-CAR7 and received a second bone marrow transplant to restore her immune function. Six months post-transplant, her cancer remains undetectable.
See More Design Blog Posts from Axxiem:
New philanthropic venture provides free open access software to help university scientist remain on the cutting edge of research A new 40-million-dollar philanthropic initiative named “Virtual institute for Scientific Software” was created by Eric Schmidt (former Google CEO) and Wendy Read more
The CORD7 Mutation Found in Humans Increases Verbal IQ and Working Memory Intelligence in its rawest form is not equally distributed among the human population. While many can critically think and problem solve, others lack these skillsets. However, what is Read more
In Vitro Neurons Learn and Exhibit Sentience Artificial Intelligence (AI) has long been touted as the next revolution in deep machine learning and the solution to solving mathematical equations that have perplexed mathematicians and physicists for decades. While the computing Read more