Genetic diseases are disorders stemming from mutations in an individual's DNA sequence. These conditions can be a result of mutations in a single or multiple genes. An example of a genetic disease is cystic fibrosis: an inherited, progressive disease that affects the respiratory system.
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Date: July 28, 2021 Time: 8:00am PDT Breast cancer is the most common form of cancer in women. It is estimated that 1 out of 8 women in the US will develop invasive breast cancer during her...
Genome editing technologies have enabled the precise manipulation of DNA sequences at targeted sites to achieve therapeutic effects. Engineered endonucleases like CRISPR-Cas9 institute site-...
Learning Objectives: 1. Background introduction to AAV gene therapy 2. Novel methods to build AAV capsid libraries for in vivo screening through rational design and directed evolution 3. Tis...
Though AsCas12a fills a crucial gap in the current genome editing toolbox, it exhibits relatively poor editing efficiency, restricting its overall utility. In collaboration with Integrated D...
Human T cells are critical effectors of immune protection from infections, autoimmune pathology, and cancer immunotherapy. We use CRISPR-mediated gene editing in primary human T cells to sys...
Lupus anticoagulants and antiphospholipid syndrome: Resolving confusion, providing new guideline updates...