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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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Scientists discover small RNA that regulates bacterial infection

The Pharma Data

Scientists discover small RNA that regulates bacterial infection People with weakened immune systems are at constant risk of infection. But the bacteria can sometimes change their behavior and enter the bloodstream, causing chronic localized infections to become acute and potentially fatal. aeruginosa infections.

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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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Gene Switch: A Novel Platform for Switching Genes On and Off

Roots Analysis

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. coli bacteria, as well as lambda bacteriophage, can adapt to the alterations in the composition of their nutrient medium.

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Scientists engineer safe, virus-resistant E coli for research

Drug Discovery World

In a step forward for genetic engineering and synthetic biology, US researchers have modified E coli bacteria to be immune to infection by all natural viruses tested so far. The team used two safeguard methods to prevent the bacteria and their modified genes from escaping into the wild.

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Johns Hopkins Researchers Identify CRISPR Dimmer

The Pharma Data

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. A CRISPR-Cas9 system has two components: the Cas9 guide RNA that directs the system to edit a particular gene, and the CRISPR “scissors” that make the cut.

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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. mRNA is a molecule made up of a single strand of ribonucleic acid (RNA). Reece Armstrong explores the potential and future opportunities for mRNA-based therapies.

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