DNA, Gene Editing and Genetic Disease - Clinical Research Informer

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Gene Editing

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Moderna partners with Life Edit for mRNA gene editing therapies

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. The company’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

Gene Editing

Gene Editing Gene In-Vivo Gene Therapy

Gene editing DNA deletion sizes reduced with new fusion method developed at WFIRM

Scienmag

MAY 2, 2022

WINSTON-SALEM, NC – May 2, 2022 — Wake Forest Institute for Regenerative Medicine (WFIRM) scientists working on CRISPR/Cas9-mediated gene editing technology have developed a method to increase efficiency of editing while minimizing DNA deletion sizes, a key step toward developing gene editing therapies to treat genetic diseases.

Gene Editing

Gene Editing DNA Gene Development

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Trending Sources

STAT+: Ahead of genome summit in London, questions linger about CRISPR baby scandal

STAT News

MARCH 3, 2023

That technology, which enables scientists to easily excise, alter, or replace specific sections of DNA, was awarded the 2020 Nobel Prize for Chemistry. Continue to STAT+ to read the full story…

Genome

Genome Genomics Gene Editing Genetic Disease

Hopewell Therapeutics raises funds for genomic medicines development

Pharmaceutical Technology

JUNE 8, 2023

The ttLNP platform can deliver a range of high-impact genomic payloads, including gene editing, siRNA, mRNA, DNA and gene-writing apparatus, to tissues, cells and organs. The development will be advanced through both internal pipeline programmes and external collaborations.

Genome

Genome Genomics Medicine Development

Generating Over a Billion Cells with CRISPR for Next Generation Cell Therapies

XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Harnessing the Cellular Engineering Potential of CRISPR.

Gene Editing

Gene Editing DNA Gene Genome

Gene Therapy and Pharmacokinetics

Camargo

JULY 27, 2021

It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy. Additionally, gene editing allows us either to remove or to modify harmful genes.

Gene Therapy

Gene Therapy Gene Gene Editing Genetics

STAT+: Pharmalittle: CRISPR-based drug shows promise in preliminary study; FDA user fee program prompts fresh worries over conflicts

STAT News

SEPTEMBER 16, 2022

Enjoy, and see you soon … Intellia Therapeutics reported that the first six patients to receive its CRISPR-based treatment for a genetic swelling disorder have safely had small, corrective changes made to dysfunctional DNA inside their liver cells , STAT writes. Continue to STAT+ to read the full story…

Gene Editing

Gene Editing Genetic Disease Genetics Protein

Asklepios partners with ReCode on gene-editing platform

Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.

Gene Editing

Gene Editing Gene Gene Therapy Genetics

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

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. pyogenes dCas9.

DNA

DNA Gene Gene Silencing Genome

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

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

DNA

DNA Gene Gene Silencing Genome

Why genomic healthcare data matters in the development of new therapies

Drug Discovery World

NOVEMBER 7, 2022

From a general healthcare perspective, access to this sort of data is invaluable, but this is particularly noteworthy in that DNA tests have historically been limited in these regions on account of cost and availability of practitioners, services and resources.

Genome

Genome Genomics Development Genetics

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

DECEMBER 13, 2023

The CRISPR system is dubbed to be molecular “scissors” that can cut out parts of genes that are abnormal, deactivating or replacing them with new strands of normal DNA. This has tremendous potential for curing diseases that are caused by faulty/mutated genes. Both therapies will be available in early 2024.

Gene Therapy

Gene Therapy FDA Approval Gene In-Vivo

CRISPR therapies targeting the next breakthrough in oncology

pharmaphorum

APRIL 20, 2022

In 2012, Jennifer Doudna and Emmanuelle Charpentier published a paper in Science where they outlined isolating the components of the CRISPR-Cas9 system and demonstrated how it could be used to cut specific sites in isolated DNA. The publication and their work eventually led to the pair being awarded the Nobel Prize in Chemistry in 2020.

Gene Editing

Gene Editing DNA Gene Genetics

Key Trends in the Life Sciences to Look Forward to in 2022

XTalks

DECEMBER 22, 2021

The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference. RNA-based treatments include RNA interference (RNAi) with double-stranded RNA molecules, or small interfering RNA (siRNA) involving single-stranded antisense oligonucleotides (ASOs).

Life Science

Life Science RNA Vaccination Vaccine

Moderna partners with Life Edit for mRNA gene editing therapies

Gene editing DNA deletion sizes reduced with new fusion method developed at WFIRM

Trending Sources

STAT+: Ahead of genome summit in London, questions linger about CRISPR baby scandal

Hopewell Therapeutics raises funds for genomic medicines development

Generating Over a Billion Cells with CRISPR for Next Generation Cell Therapies

Gene Therapy and Pharmacokinetics

STAT+: Pharmalittle: CRISPR-based drug shows promise in preliminary study; FDA user fee program prompts fresh worries over conflicts

Asklepios partners with ReCode on gene-editing platform

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

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

Why genomic healthcare data matters in the development of new therapies

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

CRISPR therapies targeting the next breakthrough in oncology

Key Trends in the Life Sciences to Look Forward to in 2022

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