Sponsor: TriLink BioTechnologiesDuring this presentation, which is part of TriLink BioTechnologies gene editing and cell therapy webinar series, Dr. Gaudelli will discuss how her group has transformed off-the-shelf base editors from a technology developed in academia to a promising therapeutic tool through engineering, screening, and directed evolution.
Firm is investigating hematopoietic stem cell gene therapies for Wiskott Aldrich syndrome and transfusion-dependent beta thalassemia.
Sponsor: Horizon DiscoveryIn this GEN webinar, sponsored by Horizon Discovery, we will provide a field overview of how iPSCs, coupled with genome editing, hold the potential to expand our understanding of human diseases further, and facilitate the development of new therapies.
A recently discovered hypercompact CRISPR enzyme found only in huge bacteriophages, and known as CRISPR-CasΦ, is functional in human and plant cells, a new study reported. CRISPR-CasΦ provides a powerful new tool in the CRISPR genome editing toolbox not only because it can fit within tiny vectors, but also because it can target a wider range of genetic sequences compared to Cas9 and Cas12.
New cytosine base editors (CBEs) achieve high targeting fidelity, showing minimum genome- and transcriptome-wide off-target effects. When engineering the CBEs, which are called APOBEC3G-nCas9 (A3G) base editors, scientists introduced mutations that allow them to minimize the conversion of so-called bystander cytosines (Cs). Specifically, the A3G base editors edit only the second of consecutive Cs.
Spider silk has many qualities that could be useful in everything from drug delivery systems to clothing. However, large quantities are required for such applications. Given that spiders naturally make small amounts of it, researchers are seeking new mechanisms for production. Now, scientists have produced spider silk using photosynthetic bacteria, creating the potential for the production of bulk amounts of the biomaterial.
A team of collaborating Howard Hughes Medical Institute (HHMI) scientists has developed what they suggest is the first precision gene editing tool for mitochondrial DNA. The CRISPR-free technology ex-ploits a deaminase toxin that is active against double-stranded DNA. To develop the mitochondrial gene editor the researchers split the deaminase into two harmless halves, which they fused to DNA-targeting proteins. The two halves recombine at the target site on the mitochondrial DNA, where the activity of complete deaminase is restored.
Using a genome-wide loss-of-function CRISPR screen, Salk Institute scientists identified genes that regulate Foxp3 in mouse primary Treg cells. Foxp3, a transcription factor crucial to the development and function of Treg cells, was found to be influenced particularly strongly by the Brd9-containing non-canonical BAF complex. Moreover, Brd9 deficiency in Treg exacerbates inflammatory bowel disease and enhances antitumor immunity.
The recent report of two deaths in the gene therapy company Audentes’ trial for children with XLMTM lead to an investigation with the help of FDA regulators to determine whether this was related to treatment with a high-dose AAV therapy. It’s important in pivotal moments like this that we quickly and calmly sort out what happened and put in place safeguards to prevent this from happening again. Patient trust in our therapies is paramount—without it we have nothing. Here is what we know so far.
There’s more to CRISPR screening than knockouts. There’s inhibition and activation—and multiplexing, too. Besides being more nuanced, the new approaches can reach once-inaccessible genomic regions.
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