Gene Expression, Gene Therapy, Protein and RNA

Gene Therapy and Pharmacokinetics

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

Gene Therapy

Gene Therapy Gene Gene Editing Genetics

Gene expression delivery tool ‘slides’ instructions into cells

Drug Discovery World

NOVEMBER 1, 2022

Researchers say they have successfully used a cell’s natural process for making proteins to ‘slide’ genetic instructions into a cell and produce critical proteins missing from those cells. . Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

Gene Expression

Gene Expression Gene Packaging Packaging

Turning science into business: Amplifying mRNA by targeting regRNAs

Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. DS: You describe your therapeutics as RNA Amplifiers. How do they work?

Gene Expression

Gene Expression Gene Genetic Disease RNA

The future outlook for mRNA therapies

Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. We can see this versatility in the various treatments that are currently being researched.

RNA

RNA Protein Vaccination Vaccine

Gene Switch: A Novel Platform for Switching Genes On and Off

Roots Analysis

AUGUST 31, 2023

Further, the expression of any gene is dependent on the rate at which it is transcribed into mRNA and translated into proteins. There are various regulatory proteins or transcription factors that are responsible for affecting the transcription rate.

Gene

Gene Gene Therapy Gene Expression Regulation

What spatial biology can tell us about disease and drug discovery

Drug Discovery World

OCTOBER 17, 2023

Today, spatial omics technologies enable imaging-based identification of cells in their native tissue context while simultaneously detecting hundreds of RNAs or tens of protein markers in the same cells. Analysing spatial changes during disease progression or treatment response is key to fully understand pathologies.

Gene Expression

Gene Expression Drugs Protein Gene

Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder

Delveinsight

JANUARY 18, 2021

However, DelveInsight’s Angelman syndrome market analysis report estimates that several approaches focused primarily on the Gene Therapies, Topoisomerase Inhibitors, Cannabidiol, Protein Phosphatase 2A inhibitor, among others are under development to find a cure for the Angelman syndrome.

Marketing

Marketing Gene Drugs Protein

CRISPR breakthroughs: New solutions for common diseases

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. CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg. parvovirus B19, Flaviviridae, Ebola, and Coronaviridae), DNA viruses (e.g.,

DNA

DNA Genome Genomics RNA

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

MAY 4, 2021

These modifications regulate gene expression without changing the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. It’s a great tool for controlling gene expression.”.

DNA

DNA Gene Gene Silencing Genome

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

XTalks

MAY 4, 2021

These modifications regulate gene expression without altering the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. It’s a great tool for controlling gene expression.”.

DNA

DNA Gene Gene Silencing Genome

Delivering on the promise of gene editing

Drug Discovery World

DECEMBER 15, 2022

Potential solutions: For CRISPR systems, the likelihood of off-target editing has been shown to be influenced by a variety of factors, ranging from the design of the gRNA 1 and Cas9 protein 2 , to the targeted cell type 3 , to name a few examples. DNA targeting specificity of RNA-guided Cas9 nucleases . In one example, Choi et al.

Gene Editing

Gene Editing Gene In-Vivo Genome

Clinical Catch-Up: December 21-25 | BioSpace

The Pharma Data

DECEMBER 27, 2020

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. The drug is a topical ointment applied to the lower lid to address the build-up and shedding of proteins at the opening of the Meibomian gland. The drug is an autologous IL13Ralpha2-CAR-T cell product.

Trials

Trials Antibody Gene Therapy Clinical Trials

The use of base editing in stem-cell based therapies

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

Gene Editing Gene Engineer DNA

2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences

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

Life Science Vaccination Vaccine Gene

Clinical Research Informer

Gene Therapy and Pharmacokinetics

Gene expression delivery tool ‘slides’ instructions into cells

Trending Sources

Turning science into business: Amplifying mRNA by targeting regRNAs

The future outlook for mRNA therapies

Gene Switch: A Novel Platform for Switching Genes On and Off

What spatial biology can tell us about disease and drug discovery

Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder

CRISPR breakthroughs: New solutions for common diseases

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

Delivering on the promise of gene editing

Clinical Catch-Up: December 21-25 | BioSpace

The use of base editing in stem-cell based therapies

2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences

Stay Connected