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Epigenetic editing: the next generation of genetic medicine

Drug Target Review

The reasons for this are multifaceted, including concerns over the safety of directly altering DNA sequences and subsequent regulatory restrictions that have arisen as a result. The epigenome (meaning ‘above the genome’) is a system of reversible marks regulating how the DNA is read, translated and used.

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DNA Analysis Finds New Target for Diabetes Drugs

NIH Director's Blog: Drug Development

Credit: Jane Ades, National Human Genome Research Institute, NIH Type 2 diabetes (T2D) tends to run in families, and over the last five years the application of genomic technologies has led to discovery of more than 60 specific DNA variants that contribute to risk. Nat Rev Drug Discov. 2013 Aug;12(8):581-94. [3]

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Cas9 ancestor engineered into a compact genome editing tool

Broad Institute

Cas9 ancestor engineered into a compact genome editing tool By Tom Ulrich May 14, 2025 Breadcrumb Home Cas9 ancestor engineered into a compact genome editing tool Researchers adapt a compact RNA-guided enzyme from bacteria for a variety of DNA editing tasks in human cells. Like Cas9, IscB enzymes cut DNA at sites specified by an RNA guide.

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Navigating the challenges and opportunities of AI in drug development and personalised medicine

Drug Target Review

I think it is astonishing that The Human Genome Project was completed exactly 20 years ago and DNA was discovered by Franklin and published by Watson and Crick exactly 70 years ago, yet that 10 percent success rate has still not improved. This means that we are not doing something right.

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New insights into the role of viral capsids in gene therapy safety

Drug Target Review

2 Structure and function of AAV capsids in gene therapy Wild-type AAV is composed of a protein shell (capsid) that contains a single-stranded DNA genome encoding proteins involved in viral replication, structure and assembly. Therefore, vectors based on these serotypes may enable targeted gene delivery and optimal transduction efficiency.

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Tonix Pharmaceuticals Enrolls First Participant in the PRECISION Study, an Observational Study to Facilitate Development of Precision Medicine Techniques for COVID-19 Vaccines and Therapeutics

The Pharma Data

Assistant Professor of Medical Sciences at Columbia University Vagelos College of Physicians and Surgeons, is designed to generate DNA aptamer-based anti-idiotypes to selected monoclonal antibodies identified in Dr. Ilya Trakht’s study. The study led by Dr. Sergei Rudchenko, Ph.D.,

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Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

Broad Institute

Related groups Liu Group Related news New CRISPR genome editing system offers a wide range of versatility in human cells Researchers extend power of gene editing by developing a new class of DNA base editors In May 2025, researchers announced that K.J. Some of these treatments, like K.J.’s, He needed a personalized, one-of-a-kind therapy.

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