CRISPR & Genetic Engineering News and Discussions

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Microbiome studies help explore treatments for genetic disorders
https://phys.org/news/2024-05-microbiom ... rders.html
by University of Trento

A collaboration has led to the identification, in a bacterium of the intestine, of new CRISPR-Cas9 molecules that could have a clinical potential to treat genetic diseases such as retinitis pigmentosa, through sub-retinal injections. Anna Cereseto and Nicola Segata of the Department of Cellular, Computational and Integrative Biology of the University of Trento have joined forces and combined their expertise to develop new therapies for the treatment of genetic diseases.

A study, with Anna Cereseto and Nicola Segata as corresponding and senior authors, has been published in Nature Communications.

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Pioneering gene therapy restores UK girl's hearing

7 hours ago

A UK girl born deaf can now hear unaided, after a groundbreaking gene-therapy treatment.

Opal Sandy was treated shortly before her first birthday - and six months on, can hear sounds as soft as a whisper and is starting to talk, saying words such as "Mama", "Dada" and "uh-oh".

Given as an infusion into the ear, the therapy replaces faulty DNA causing her type of inherited deafness.

Opal is part of a trial recruiting patients in the UK, US and Spain.

Doctors in other countries, including China, are also exploring very similar treatments for the Otof gene mutation Opal has.

https://www.bbc.co.uk/news/health-68921561

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CRISPR restores some vision to blind patients in clinical trial
By Michael Irving
May 13, 2024

CRISPR gene-editing has improved the vision of patients with an inherited form of blindness, according to results of a Phase 1/2 clinical trial. The results not only give new hope to patients with the condition, but show that CRISPR could be put to use in humans to treat a range of conditions.

Leber Congenital Amaurosis (LCA) is a rare condition that affects about one in every 40,000 newborns. Those affected have a genetic mutation that results in severely reduced vision, leading to complete blindness in about a third of patients. Currently, there are no FDA-approved treatment options at all.

But that might be about to change. The BRILLIANCE trial has been investigating using CRISPR to edit the CEP290 gene, one of the main culprits behind LCA, in 14 patients. The gene-editing treatment is delivered directly to the light-sensitive cells behind the retina, making this the first time CRISPR had been put to work directly in the human body.

https://newatlas.com/medical/crispr-res ... cal-trial/