Making deliberate alterations to the DNA sequence at targeted locations in the genome. This can include deleting, inserting or replacing sections of DNA. Several approaches can be used to achieve these changes including zinc finger nucleases (ZFNs), TALENs, CRISPR/Cas and base editing.
Dr Jess Buxton attended PET's most recent event, which discussed how to improve diagnosis, treatment and support for people affected by rare and inherited metabolic diseases...
The UK government has committed to accelerating the development and approval of treatments for rare diseases, through major reforms to current regulation...
A peer-reviewed paper reports proof-of-concept that a genome-edited pig liver can successfully support liver function in human patients...
Professor of stem cell science, Dusko Ilic, discusses concerns raised by the plans of a US-based startup – the (new) Manhattan Project – to create genome-edited babies…
CRISPR/Cas9 has been used to identify genome editing targets that significantly enhance the efficiency of a targeted therapy for myeloma, a type of cancer...
An artificial intelligence tool for helping to create CRISPR workflows can enable beginners to design successful experiments in a single attempt...
Knocking out a single gene on the Y chromosome has been found to cause infertility in male mice...
Base editing has been used to correct the mutation causing late-onset Tay-Sachs disease in mice and human cells...
Side effects of viral-delivered gene therapies have been elucidated using human stem-cell derived kidney organoids...
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