pharmaphorum

JANUARY 29, 2021

This growth reflects the continued and increasing interest in gene therapy – and with stock prices rising for editing companies across the board, Macrae says there has never been a more interesting time to be in genomic medicine. Zinc fingers are the most common control gene in the body,” Macrae explains. “We Macrae explains. “So

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Gene Editing Gene Gene Therapy In-Vivo 145

pharmaphorum

FEBRUARY 22, 2021

Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. .

Gene Therapy 52

Gene Therapy Gene Gene Sequencing Gene Expression 52

XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. BacTRL Gene Therapy Platform. Related: Red Meat Allergy Test Gets FDA Clearance. “We

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Clinical Trials Clinical Trials Vaccination Vaccine 75

XTalks

MARCH 13, 2024

There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease. The Glaucoma Foundation has funded studies focusing on two specific areas within gene therapy.

Gene Therapy 107

Gene Therapy Gene Life Science DNA 107

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. It’s a great tool for controlling gene expression.”.

DNA 97

DNA Gene Gene Silencing Genomics 97

The Pharma Data

JULY 12, 2021

“This is mostly because drugs that work perfectly in preclinical inbred models, such as laboratory mice, that are genetically or otherwise identical to each other, don’t translate to patients in the clinic, where each individual and their disease is unique. Nature Communications, 2021. doi: 10.1038/s41467-021-24470-5.

Drugs 52

Drugs Gene Expression Big Data Gene 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 without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA 52

DNA Gene Gene Silencing Genomics 52

Roots Analysis

JANUARY 14, 2024

Usually, the desired gene, such as human insulin gene, when inserted into the plasmid of the host cell uses transcriptional and translational machinery of the host to express itself. It is worth mentioning that in vitro gene expression requires a suitable host for the production of a specific gene product.

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Contract Manufacturing Protein Manufacturing Production 40

Delveinsight

JANUARY 18, 2021

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation.

Marketing 52

Marketing Gene Drugs Protein 52

The Pharma Data

DECEMBER 27, 2020

STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 gene expression. RESTORE-1 is a Phase II trial of an intracerebral AAV-based gene therapy for Parkinson’s disease.

Trials 52

Trials Antibody Gene Therapy Gene 52

The Pharma Data

MAY 15, 2021

I was recently reminded of my journey out of the HCT nightmare as I watched a documentary about gene therapy. Gene therapy is a huge deal – some gene therapies are already achieving jaw-dropping results for people with previously untreatable illnesses. It’s fascinating stuff. They’d beaten HCT.

Bacteria 52

Bacteria Gene Gene Therapy Gene Expression 52

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

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Life Science Vaccination Vaccine Gene 109