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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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SNIPR targets antibiotic resistance with CRISPR-based gene therapy trial

Pharmaceutical Technology

The Danish CRISPR biotech SNIPR BIOME has released the first glimpse at the safety profile for SNIPR001, a gene therapy intended to target antibiotic resistance. CRISPR or Clustered Regularly Interspaced Short Palindromic Repeats, provides the opportunity to target and modify specific DNA sequences.

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Guide to using AAV vectors in gene therapy

Drug Discovery World

In 2023, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers. Why are those important?

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Guide to using AAV vectors in gene therapy

Drug Discovery World

Earlier this year, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

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New CRISPR-Based Tool Called PASTE Gene Editing Inserts Large DNA Sequences at Desired Sites

XTalks

Developed by MIT researchers Jonathan Gootenberg and Omar Abudayyeh, PASTE (Programmable Addition via Site-specific Targeting Elements) gene editing technology can insert genes as long as 36,000 DNA base pairs to liver cells in mice as well as several types of human cells.

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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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CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. parvovirus B19, Flaviviridae, Ebola, and Coronaviridae), DNA viruses (e.g., Herpesviridae, Polyomaviridae, and Papillomaviridae), bacteria (e.g.,

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