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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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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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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. In bacteria with unaltered tracr-L, levels of CRISPR-related genes were low. The authors found that tracr-L redirects Cas9 in S.

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Key Trends in the Life Sciences to Look Forward to in 2022

XTalks

The RNA Revolution: From mRNA Vaccines to RNA Editing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. RNA technology is not new nor has its potential been surprising. RNA in the Making. So why did this perceived RNA ‘revolution’ take so long?

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The doors CRISPR libraries have and will open in phenotypic drug screening 

Drug Discovery World

The CRISPR-Cas9 system, awarded the Nobel Prize in Chemistry in 2020 for its applications for gene editing, was initially encountered in the bacterial immune system. Bacterial RNA is replaced by a synthetic guide RNA (gRNA) that can recognise a specific target DNA sequence. CRISPR libraries: Why do we need them?

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CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

Since its discovery in 2012, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology has revolutionised the biomedicine and cell and gene therapy fields, providing a versatile tool for precise and efficient genome editing. CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg.

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Top 30 Pharma and Biotech Companies in 2023: Statistics and Trends

XTalks

Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. 2) Botox Therapeutic/Cosmetic Botox, or botulinum neurotoxin, is a neurotoxic protein produced by the Clostridium botulinum bacteria. But all three drugs have new competitors. Both are anti-CD20 agents. billion in 2022.

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