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Gene editing: beyond the hype

pharmaphorum

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”. Zinc fingers.

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This week in drug discovery (2-6 October)  

Drug Discovery World

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs 52
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AI-designed protein awakens silenced genes, one by one

The Pharma Data

The approach will allow researchers to understand the role individual genes play in normal cell growth and development, in aging, and in such diseases as cancer, said Shiri Levy, a postdoctoral fellow in UW Institute for Stem Cell and Regenerative Medicine (ISCRM) and the lead author of the paper. Cas9 binds and uses RNA as an address-tag.

Protein 52
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Epigenetic Editing with CRISPR + ADHD in Preschoolers – Xtalks Life Science Podcast Ep. 10

XTalks

In this episode, Ayesha discusses a new tool that uses CRISPR to modulate gene expression without editing DNA sequences. Dubbed “CRISPRoff,” the technology targets the epigenome to silence genes involved in diseases, with applications in cancer, AML and other conditions with a heritable component.

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Delivering on the promise of gene editing

Drug Discovery World

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR. pyogenes dCas9.

DNA 98
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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.

Genetics 119