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Exa-Cel CRISPR Gene Therapy for Sickle Cell Disease Deemed Safe by FDA Advisory Panel

XTalks

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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Biologics – The Next Step in Revolutionary Medication

Roots Analysis

Usually, the desired gene, such as human insulin gene, when inserted into the plasmid of the host cell uses transcriptional and translational machinery of the host to express itself. It is worth mentioning that in vitro gene expression requires a suitable host for the production of a specific gene product.

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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.

DNA 98
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The evolution of assays for immuno-oncology research

Drug Discovery World

The first such research dates back to 1891, when William Coley attempted to inject heat-inactivated bacteria to treat osteosarcoma. Genetically modified T cells stimulated with IL2 are now administered as cell therapy for multiple cancer cell types with beneficial results 3,4. This continues to be the standard of care.

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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.

Gene 40
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Bacteriophage Therapy – A Revolutionary Approach Towards Multi-Resistant Bacteria

Roots Analysis

Bacteria plays a crucial role in maintaining the ecosystem balance. However, there are few species of bacteria that can cause several infectious diseases ( such as strep throat, salmonellosis, tuberculosis, whooping cough ). The overuse of antibiotics has resulted in the emergence of multi-drug-resistant bacteria.

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Genotoxicity Testing: Unlocking the Future Safety Assessment Opportunities

Roots Analysis

Genotoxicity testing refers to the evaluation of detrimental effects of chemical or physical agents on the genetic processes and related hereditary material of living cells. Mechanism of Genotoxicity / Mutagenicity The interaction of genotoxins / mutagens with the structure of DNA causes damage to the genetic material.