Bio Saga Blog - A Chronicle of Life Sciences & Informatics

https://singularityhub.com/2019/05/02/crispr-used-in-human-trials-for-the-first-time-in-the-us/amp/ CRISPR  just hit another landmark. Last week, the University of Pennsylvania (UPenn) confirmed that they have treated two cancer patients using the gene editing darling married with another biomedical wizard,  CAR-T . For now, it’s too early to tell if the treatment proved beneficial in either patient, but the team hopes to release a first batch of results in a conference or journal “ at an appropriate time .” It marks CRISPR’s first debut onto the US clinical scene, and it’s a long time coming. As early as mid-2016 the team had already received approval from the FDA to move ahead with their concept. Yet they didn’t register the trial  until early 2018 , taking careful precautions to not royally mess up CRISPR’s introduction into the biomedical mainstream. This is in stark contrast to China, which, due to laxer clinical registration rules,  kicked off its ...

In its simplest sense, we imagine that learning occurs through a series of positive and negative rewards. Some actions lead to pleasure, others to pain, and it seems reasonable to expect that people will repeat the actions with pleasurable results and avoid those that ended in pain. Yet, we all know people who aren't deterred by the idea of punishment. We all know people who never seem to learn. Could there be a physical reason, hidden in their genes? Read the rest of this post... | Read the comments on this post...... more Klein, T. (2007). Genetically Determined Differences in Learning from Errors. Science, 318 (5856), 1642-1645.

A protein related to heart disease and Alzheimer's is found to be a factor in HIV. The apolipoprotein (apo) E4 isoform has been implicated in neurodegeneration in Alzheimer's disease, cardiovascular disease, and stroke. Now, investigators at the Gladstone Institutes, the University of California, San Francisco, the University of Texas Health Science Center at San Antonio, and the Infectious Disease Clinical Research Program, Uniformed Services University, Bethesda, Maryland have shown that this troubling protein is a risk factor for AIDS progression rates and promotes entry of HIV into cells. Do you want to read more?

The US Food and Drug Administration issued a final guidance last week regarding investigational new drug applications for human gene therapy. Although gene therapy is still in the developmental stages, there is much hope for gene-based treatment and products. Because the therapy involves replacing a patient's faulty genetic material with normal genetic material,there is concern about safety in making sure replacement cells continue to function as they should. The guidance, therefore, focuses on the type of information that scientists need to consider and include in their documentation when studying these drugs and submitting them for FDA approval. Based on a draft guidance that was issued in November 2004 (69 FR 64958), the final document, Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) , provides information on what to include in an original IND. The document also applies to combinat...