pharmaphorum

JANUARY 29, 2021

‘Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Macrae explains. “So Most of them are rare mutations.”.

Gene Editing 145

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

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.

Contract Manufacturing 40

Contract Manufacturing Manufacturing Protein Bacteria 40

Roots Analysis

AUGUST 31, 2023

Gene switches can be regulatory proteins or specific DNA sequences that act to either switch on or off the expression of a gene. Basic Components of Gene Switch Gene switches are composed of noncoding DNA sequences and transcription factors.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

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 98

DNA Gene Gene Silencing Genome 98

Drug Discovery World

FEBRUARY 29, 2024

They are called: Sitting down with… PharmEnable , Is single-cell gene expression the next trend in next generation sequencing? In the second article, we hear from Dr Dina Finan, PhD, Product Manager at 10x Genomics and Dr Nick Downey, PhD, NGS Collaborations Lead at Integrated DNA Technologies.

Gene Expression 52

Gene Expression DNA Genome Genomics 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 Genome 52

Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. In one example, Choi et al.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

APRIL 30, 2024

Rare diseases are largely treated using gene therapies which employ modified viruses to bring the therapy to the disease cells. However, these therapies can cause severe adverse effects. The benefits of mRNA mRNA-based therapies have multiple benefits over existing DNA and protein platforms.

RNA 52

RNA Protein Vaccination Vaccine 52

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. parvovirus B19, Flaviviridae, Ebola, and Coronaviridae), DNA viruses (e.g., It also enables the downregulation of gene expression by directly knocking out the cytoplasmic mRNA transcripts.

DNA 98

DNA Genome Genomics RNA 98

Drug Discovery World

OCTOBER 17, 2023

RA: With spatial biology being cross-disciplinary – in that it uses techniques in both gene expression analysis and immunofluorescence – is expertise in the field limited? Analysing spatial changes during disease progression or treatment response is key to fully understand pathologies. Let’s take CAR T cells as an example.

Gene Expression 52

Gene Expression Drugs Protein Gene 52

Drug Discovery World

JANUARY 23, 2023

Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2. Base editing uses a guide RNA in partnership with a nickase version of Cas9, mutated to cut only a single strand of DNA, along with a deaminase enzyme to enable alteration of a single nucleotide 23,24.

Gene Editing 52

Gene Editing Gene Engineer DNA 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 Clinical Trials 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.

Life Science 111

Life Science Vaccination Vaccine Gene 111