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Gene Therapy and Pharmacokinetics

Camargo

It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy. Additionally, gene editing allows us either to remove or to modify harmful genes.

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Turning science into business: Amplifying mRNA by targeting regRNAs

Drug Discovery World

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What diseases are you currently targeting? Can you give some examples?

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Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”.

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

XTalks

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

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Molecular Diagnostic Tests: The Unsung Hero of Precise Clinical Decision Making

Roots Analysis

These diagnostic solutions are essential for detecting and monitoring diseases, identifying genetic abnormalities, and guiding personalized treatment plans. Molecular diagnostic solutions are pivotal across various medical fields, including oncological disorders, infectious diseases, genetic testing, and personalized medicine.

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The evolution of the RNA therapeutics landscape 

Drug Discovery World

For decades, the life sciences community held the belief that RNA was merely the intermediary messenger (mRNA) between DNA and protein, a dogma that has been the foundation of biology. RNA interference: silencing gene expression The second category of RNA therapeutics is a class that has also yielded marketable drugs in recent years.

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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. We believe it can now do the same for rare diseases.” “We We have shown that mRNA holds an unprecedented therapeutic potential for incurable genetic diseases, in particular liver conditions.

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