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In addition, gene-editing technologies continue to advance in precision and ease of use, allowing families to treat and ultimately cure hundreds of inheritable genetic diseases.
This metatrend is driven by the convergence of: various biotechnologies (CRISPR, Gene Therapy), genome sequencing, and artificial intelligence.
Gene editing holds promise for the treatment of cancers that are driven by well-characterised molecular alterations. A study now provides a proof of concept for the feasibility of in vivo gene editing to correct TERT mutations in glioblastoma, providing a platform for the direct manipulation of genetic alterations to reduce tumour growth.
A Cambridge-based biotech company that is creating a colony of “human” mice is locked in a Tom and Jerry-style fight with a US pharmaceutical giant.
Kymab, which is backed by the Bill and Melinda Gates Foundation and counts fund manager Neil Woodford among its investors, is seeking permission to appeal to the Supreme Court in a row over patents. It follows a ruling by the Court of Appeal earlier this year that Kymab infringed a patent belonging to US company Regeneron. A previous judgment ruled that Regeneron’s patent was insufficient.
The legal tussle relates to Kymab’s “Kymouse” work, in which it manipulates the genome of mice. Kymab removes the genes that make antibodies in mice and replaces them with human antibody genes. This means that.
When tested in mice, the molecule, dubbed halicin, effectively treated the gastrointestinal bug Clostridium difficile (C. diff), a common killer of hospitalized patients, and another type of drug-resistant bacteria that often causes infections in the blood, urinary tract, and lungs.
The most surprising feature of the molecule? It is structurally distinct from existing antibiotics, the researchers said. It was found in a drug-repurposing database where it was initially identified as a possible treatment for diabetes, a feat that showcases the power of machine learning to support discovery efforts.
