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Gene Therapy and Pharmacokinetics

Camargo

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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A startup emerges with $125M and plans to edit the epigenome

Bio Pharma Dive

Chroma Medicine's launch is the latest step in a decadeslong quest by drugmakers to capitalize on research into epigenetics, a way of controlling gene expression without altering DNA.

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New facility boosts UK’s cell and gene therapy manufacturing capacity

Pharmaceutical Technology

On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UK’s ability to develop and manufacture cell and gene therapies. In the past, researchers have often had to seek help from outside the UK, thus delaying clinical trials and patient access.

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New CRISPR-Based Tool Called PASTE Gene Editing Inserts Large DNA Sequences at Desired Sites

XTalks

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.

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Researchers speed identification of DNA regions that regulate gene expression

Scienmag

Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Jude Children’s Research Hospital St. Credit: St.

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Researchers discover key role of DNA methylation in Alzheimer's disease

Medical Xpress

Mount Sinai researchers have published a study in Alzheimer's & Dementia: The Journal of the Alzheimer's Association that sheds new light on the role of DNA methylation in Alzheimer's disease (AD).

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UK scientists say they have found cancer driver in junk DNA

pharmaphorum

It has suspected for many years that some diseases may be linked to non-coding or ‘junk’ DNA, but the mechanism behind the pathology hasn’t been worked out. Junk DNA is a term used to describe the 97% of the genetic sequence in human cells found between the 3% coding for our 20,000 genes, once thought to be inert.

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