Cutting-edge research conducted by experts at Michigan Medicine, the Children’s Hospital of Philadelphia, and Penn Medicine is revolutionising the approach to treating blood disorders like sickle cell anaemia through innovative gene therapy methods.
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Sickle cell anaemia
In an exclusive interview, Dr Neville Sanjana, Associate Professor of biology at NYU and a core faculty member at the New York Genome Center, discusses the breakthrough study on STING-seq.
In the unmet need for effective treatments to alleviate suffering and prevent premature death across the spectrum of genetic diseases, Brent Warner, Poseida Therapeutics, shares that the ability to edit DNA holds hope for patients currently experiencing incurable genetic diseases and has spurred ongoing efforts to develop and improve gene…
This article highlights a new approach to address human genetics, using STING-seq which provides roadmap to identify variants and genes, enabling deeper understanding of the noncoding genome and targets for therapies
Researchers have published a step-by-step protocol on how to produce millions of mature human cells in a chimeric mouse embryo.
In October this year, Emmanuelle Charpentier and Jennifer Doudna, the two scientists who pioneered the revolutionary gene-editing technology CRISPR, were awarded the Nobel Prize in Chemistry. Here, Pushpanathan Muthuirulan discusses the potential for this technology and the importance of using it safely, ethically and responsibly.
A team of researchers has discovered a Cas9 enzyme that can target almost half of the locations on the genome, significantly widening its potential application.