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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. This treatment activates the OTC gene so that ammonia can be removed from the blood. In CAMP4’s approach, regRNAs are drugged with antisense oligonucleotides to control the direction of gene expression.

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Gene Therapy and Pharmacokinetics

Camargo

Research and development in the area is currently growing at a fast rate, and the National Institute of Health reports hundreds of clinical trials to test gene therapies for different genetic diseases, immune system disorders, oncology treatments, neurogenerative diseases, infectious diseases, and more.

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Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

These modifications regulate gene expression without changing the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. “Now pyogenes dCas9.

DNA 98
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Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

XTalks

These modifications regulate gene expression without altering the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. “Now pyogenes dCas9.

DNA 52
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Clinical Catch-Up: December 21-25 | BioSpace

The Pharma Data

STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 gene expression. GA Depot is a long-acting injection of the approved Glatiramer Acetate products (Copaxone and its generic formulations).

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