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Lipid nanoparticles and mRNA used to treat hereditary blindness

Drug Discovery World

The study, led by OSU Associate Professor of Pharmaceutical Sciences Gaurav Sahay, Oregon State doctoral student Marco Herrera-Barrera, and Oregon Health & Science University Assistant Professor of Ophthalmology Renee Ryals, was published in Science Advances. .

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How to advance AAV-based gene therapies

Drug Discovery World

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

Therapies 162
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Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

Lumping gene and cell therapies together isn’t very helpful – the cell-based ones are mostly doctored T cells to treat cancer. million DNA bases. The DMD gene therapy delivers a shortened version of the dystrophin gene, just 4,558 DNA bases. He was treated on a Monday and died that Friday, as his organs shut down.

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Not all neoantigens are created equal

Drug Target Review

During the process of transformation from a normal cell into a cancer cell, a cell acquires a series of changes, or mutations, in its DNA. But DNA mutations can also result in changes to the proteins that are displayed on the surface of the cancer cell. Our immune system helps us to fight off infections and cancer.

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Answering Your Qs on the New COVID Vaccines

The Pharma Data

The COVID-19 vaccines work by teaching the immune system to protect against the virus, experts said. Neither of the two vaccines approved in the United States — made by Pfizer-BioNTech and Moderna — contains the live virus. “That means it never converts into DNA,” Dr. M.

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The democratisation of cell and gene therapy

Drug Discovery World

DNA purification “Plasmid DNA is an incredibly important part of molecular biology”, says Hummersone. We are now looking at larger plasmid DNA sequences to carry more genetic material, and so we can get a more delicate endpoint with a much larger plasmid DNA sequence. That’s not the case in CGT.”

Therapies 273
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Defense-Forward Biosecurity

Codon

Allison Berke makes the case for real-time DNA sequencing and AI tools to detect pathogens before they spread widely. Doctors in training are told that when they hear hoofbeats, they should think horses, not zebras; rare diseases are the exception, not the rule. After copying the DNA to form a big pool, each piece is sequenced.

DNA 84