Drug Discovery World

OCTOBER 4, 2022

BV-101 is an adeno- associated virus (AAV) gene therapy vector that simultaneously addresses the metabolic dysfunction of diseased neurons and contributes to the clearance of the mutant huntingtin protein. In the UK, effort has been made to scale-up the country’s cell and gene therapy manufacturing capabilities.

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XTalks

MAY 4, 2021

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Genes can be switched on with a complementary tool called CRISPRon that has also been described in the paper. Epigenetic Editing with CRISPR.

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DNA Gene Gene Silencing Genome 98

Drug Discovery World

AUGUST 1, 2023

1 August was first chosen as World RNA Day in 2018 as a play on AUG (adenine, uracil and guanine), a triple sequence of RNA (called a codon) that initiates protein synthesis by the cell. Since then, it has been observed to publicise the importance of this molecule in the generation of proteins in the body.

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RNA Drugs Gene Silencing Vaccination 52

XTalks

MAY 4, 2021

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Genes can be switched on with a complementary tool called CRISPRon that has also been described in the paper. Epigenome Editing with CRISPR.

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DNA Gene Gene Silencing Genome 52

The Pharma Data

APRIL 27, 2023

(Nasdaq: ALNY ) announced today positive interim results from the ongoing single ascending dose part of the Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy (CAA). Chief Medical Officer of Alnylam. “We

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Roots Analysis

OCTOBER 22, 2023

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein.

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RNA Protein Genetics Gene Expression 40

Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector. Engineering antigen-specific primary human NK cells against HER-2 positive carcinomas. 3002842.

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Gene Editing Gene Engineer DNA 52