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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

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Johns Hopkins Researchers Identify CRISPR Dimmer

The Pharma Data

In the paper, researchers from Johns Hopkins University and elsewhere found a natural long-form transactivating CRISPR RNA (tracr-L) in Streptococcus pyogenes that functions to downregulate its endogenous CRISPR-Cas9 system. But altering the tracr-L with genetic engineering to make it function more like a guide RNA increased CRISPR-Cas9 cuts.

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mRNA Synthesis: ManufacturingProcess of Modern Revolutionary Molecule

Roots Analysis

It transfers genetic information form to DNA to ribosomes, a specialized structure, or organelle, which decodes genetic information into a protein. With the help of genetic engineering, synthetic mRNAs can express proteins, as they structurally resemble a natural mRNA.

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Risk Assessment for use of Engineered Genetic Materials in Clinical Research

Advarra

This trend came to the forefront during the COVID-19 pandemic, as the three most popular vaccines developed in response to the coronavirus contained engineered genetic materials in the form of mRNA or a genetically engineered virus.

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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. Cancer Immunotherapies : RNA molecules, such as siRNA, microRNA, and mRNA, have shown tremendous potential for immunomodulation and use as a cancer immunotherapy.

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Improving Study Activation Time for Gene Therapy Research

Advarra

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. When treatments involve recombinant or synthetic nucleic acids, including messenger RNA (mRNA) or viral vector vaccines, additional IBC oversight is necessary.

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Clinical Catch-Up: January 4-8 | BioSpace

The Pharma Data

ILP100 is a genetically engineered Lactobacillus that acts as a small bioreactor on site in the wound, continuously introducing a chemokine, CXCL12, into the wound tissue. The drug is an RNA interference (RNAi) therapeutic. ENPP1 deficiency is a rare, chronic, systemic, and progressive disease.