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.

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 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.

DNA 98

DNA Gene Gene Silencing Genome 98

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. — Eric Topol (@EricTopol) June 26, 2021.

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In-Vivo Gene Editing Trials Drugs 104

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.

DNA 52

DNA Gene Gene Silencing Genome 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.

RNA 52

RNA Drugs Gene Silencing Vaccination 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. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. 2016;353(6305):aaf8729. 2016;533, 420–424.

Gene Editing 52

Gene Editing Gene Engineer DNA 52