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Risk Assessment for use of Engineered Genetic Materials in Clinical Research

Advarra

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. Either way, occupational exposure to these gene delivery systems bears potential risks to the research staff.

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Clinical Catch-Up: January 4-8 | BioSpace

The Pharma Data

FBX-101 is a first-in-human AAV gene therapy. Krabbe disease is a rare and fatal pediatric leukodystrophy caused by mutations in the galactosylceramidase (GALC) gene. INZ-701 is a soluble, recombinant protein containing the extracellular domain of native human ENPP1 fused to the Fc domain of the immunoglobulin IgG1.

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

Roots Analysis

Further, the transcription factors contain two molecular domains, the DNA binding domain and the activation domain. Moreover, some transcription factors contain additional domains, such as ligand binding domain in order to interact with chemical signals.

Gene 40
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mRNA Therapeutics and mRNA Vaccines Industry: Current Scenario and Future Trends

Roots Analysis

The IVT mRNAs are structurally similar to natural mRNAs, these synthetic mRNAs can be used to express proteins through genetic engineering. Furthermore, these drug candidates can be used to develop precise and individualized therapies that allow patients to produce therapeutic proteins in their own bodies.