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Exa-Cel CRISPR Gene Therapy for Sickle Cell Disease Deemed Safe by FDA Advisory Panel

XTalks

CRISPR works as genetic scissors to edit parts of the genome. The CRISPR-Cas9 gene editing system was first discovered to be endogenous in bacteria. The companies used data from the 1,000 Genomes Project but from that, only 61 datasets made the cut to encompass the ideal patient population. “I

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Women in Science Who Have Paved the Way Forward in Genetics

XTalks

The Human Genome Project recently marked 20 years since the publication of the first full sets of human genomic sequences, an endeavor that spanned well over a decade. Today, new next-generation sequencing technologies allow for the sequencing of complex genomes within just a day or two. Emmanuelle Charpentier.

Genetics 119
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The future of genomic medicine: can it fulfil its promises?

pharmaphorum

Here he gives us a deeper look at how genomic medicine is evolving and the barriers that are preventing it from reaching its full potential. For example, we are beginning to see genomic medicine being used to treat disorders caused by a single faulty gene, so the ability to replace the gene (or the affected part of the gene) should help.

Genome 119