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In a first, children with rare genetic diseases get mitochondrial transplants from their mothers

STAT News

At a far distant point in Earth’s ancient past, two separate, single-celled life forms — an archaeon and a bacteria — became one in an act either of symbiosis or enslavement, depending on which microbiologist you ask. These capsule-shaped organelles don’t just turn oxygen and nutrients into chemical energy.

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Education and empowerment would provide more equal access to care

pharmaphorum

Ella, 28, was diagnosed with cystic fibrosis – a rare genetic disease which causes a build-up of thick, sticky mucus in the lungs, digestive tract, and sinuses – at 18 months old. This sticky mucus gets trapped and breeds infections from bacteria I inhale,” says Ella, who adds she was “terrified” of doctors as a child.

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Gene Therapy and Pharmacokinetics

Camargo

The technology of gene therapy is possible due to extensive DNA research and our resulting understanding of many diseases on the genetic level, and it encompasses several mechanisms such as introducing new genes (gene addition) and inactivating or replacing mutated genes (gene editing).

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Key Trends in the Life Sciences to Look Forward to in 2022

XTalks

This included Eli Lilly’s partnership with Netherlands-based RNA biotech ProQR Therapeutics this year to develop RNA editing-based therapies for rare genetic diseases, namely blindness-causing retinal conditions, based on the company’s proprietary Axiomer RNA editing platform technology. The deal is worth $1.5