Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Breakthrough in CRISPR delivery promises safer gene editing

Recent research in PNAS highlights advancements in non-viral, cell-specific delivery methods for CRISPR-Cas systems, emphasizing their potential to enhance research and gene therapy applications by minimizing risks associated with off-target effects and improving targeted delivery.

7 Mar 2024

Higher COVID-19 death risk found in sickle cell disease and trait patients

UdeM's Stéphanie Forté and co-researchers in Canada and Brazil have shown how people with sickle cell disease or sickle cell trait have a higher chance of dying from COVID-19 than the general population.

29 Feb 2024

Genetic studies shed light on hypertension risk but fall short of revealing new treatments

Genetic variant research in hypertension has identified numerous associations but is unlikely to yield new treatments, emphasizing the gap between scientific discovery and clinical application.

27 Feb 2024

Bio-Rad launches vericheck ddPCR replication competent lentivirus and replication competent AAV kits for cell and gene therapy production

Rapid, Sensitive, and Validated Detection of Viral Replication Genes Using Droplet Digital™ PCR During Cell and Gene Therapy Production.

From Bio-Rad Laboratories 27 Feb 2024

Breakthrough gene therapy method utilizes bird retrotransposon for safe gene insertion

The recent approval of a CRISPR-Cas9 therapy for sickle cell disease demonstrates that gene editing tools can do a superb job knocking out genes to cure hereditary disease.

20 Feb 2024

MIP Discovery closes £7M Series A financing to drive commercialization in cell and gene therapy space

Company’s synthetic affinity reagents are antibody alternatives designed to advance the speed, safety, and efficiency of manufacturing at scale, making advanced therapies more accessible to patients.

From MIP Discovery 20 Feb 2024

Researchers develop novel nanomedicine loaded with genetic material to fight HIV

Society learned about the value of mRNA during the COVID-19 pandemic when we saw scientists and medical professionals harness its power to deliver a vaccine for the virus within a year.

20 Feb 2024

City of Hope cures oldest person of blood cancer and achieves HIV remission

City of Hope, one of the largest cancer research and treatment organizations in the United States, treated the oldest person to be cured of a blood cancer and then achieve remission for HIV after receiving a blood stem cell transplant from a donor with a rare genetic mutation.

15 Feb 2024

Gene therapy reverses advanced heart failure in animal study

Heart failure remains the leading cause of mortality in the U.S. During a heart attack blood stops flowing into the heart.

13 Feb 2024

Gene editing gets a boost with new CRISPR-COPIES

CRISPR/Cas systems have undergone tremendous advancement in the past decade. These precise genome editing tools have applications ranging from transgenic crop development to gene therapy and beyond.

12 Feb 2024

GoFundMe has become a health care utility

GoFundMe started as a crowdfunding site for underwriting "ideas and dreams," and, as GoFundMe's co-founders, Andrew Ballester and Brad Damphousse, once put it, "for life's important moments."

12 Feb 2024

Never-before-documented genetic disorder causes rare lung disease

The macrophage is one of the body's most important inhabitants. Meaning "big eater" in Greek, this immune cell consumes and digests problematic elements from microbes and cancer cells to dust and debris.

8 Feb 2024

First gene therapy trial aims to restore hearing in children

The aim of this clinical trial, which has just received approval in France, is to assess the safety and efficacy of a new gene therapy drug in children aged between 6 and 31 months with profound hearing loss.

5 Feb 2024

Revvity showcases cutting-edge solutions at SLAS2024

Revvity presents its continued commitment to expanding the boundaries of human potential through science.

From Revvity 5 Feb 2024

Breakthrough recombinase technology ushers in precise, adaptable gene editing

A team of researchers at the Carl Gustav Carus Faculty of Medicine, TUD Dresden University of Technology, led by Prof. Frank Buchholz, has achieved a major breakthrough in genome editing technology.

1 Feb 2024

Gene therapy could prolong survival in some people with arrhythmogenic right ventricular cardiomyopathy

A new study in mice shows that replacement of a dysfunctional gene could prolong survival in some people with arrhythmogenic right ventricular cardiomyopathy (ARVC), a rare inherited disorder in which the muscular walls of the heart progressively weaken and put patients at risk of dangerous irregular heartbeats.

30 Jan 2024