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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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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). Formulation Considerations.

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Destiny Pharma granted award from Cystic Fibrosis Foundation  

Drug Discovery World

Cystic fibrosis (CF) is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe. MRSA is becoming more common among the pathogenic bacteria that cause lung infections in cystic fibrosis patients. It’s thought that around 25% of people with the disease are infected with MRSA.

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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.

Bacteria 105
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The future outlook for mRNA therapies

Drug Discovery World

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. Their functions such as carrying oxygen, providing structural support to cells, transmitting biological processes to cells, and fighting viruses and bacteria, are essential to human health.

RNA 52
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Key Trends in the Life Sciences to Look Forward to in 2022

XTalks

RNA therapeutics hold promise as powerful treatments for diseases, including those that are genetic-based. They can provide continuous real-time monitoring of metabolites, bacteria, hormones, glucose and other elements in these fluids. RNA Therapeutics. The deal is worth $1.5 Prior to this, Lilly struck an agreement worth $1.25