Drug Discovery World

OCTOBER 4, 2022

As it stands, there are currently 19 gene therapies approved for clinical use, alongside 18 RNA therapies and 59 non-genetically modified cell therapies, according to the Q2 report by the American Society of Cell & Gene Therapy. million by Horizon Europe which will go towards its goal of developing bioprocessing technologies. .

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Gene Therapy Drugs Gene Manufacturing 52

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

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.

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DNA Gene Gene Silencing Genome 98

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

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). Yancopoulos, M.D.,

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XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. Epigenome Editing with CRISPR.

DNA 52

DNA Gene Gene Silencing Genome 52

Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies.

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Gene Editing Gene Engineer DNA 52