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.

AI Predicts Mutations for Improved Protein Function

To design proteins with beneficial functions, researchers typically start with a natural protein possessing a desirable function, such as emitting fluorescent light.

5 Apr 2024

Metabolic Insights into Immune Cell Activation and Response

Researchers from Children's Hospital of Philadelphia identified a key metabolite in cells that helps direct immune responses and explains at a single cell level why immune cells that most efficiently recognize pathogens, vaccines, or diseased cells grow and divide faster than other cells.

18 Mar 2024

Retrotransposon-Based PRINT Technique Holds Potential for Precise 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.

22 Feb 2024

The Promise of siRNA Nanomedicine for HIV Treatment

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

21 Feb 2024

MIP Discovery Closes £7M Series a Financing to Drive Commercialization in Cell and Gene Therapy Space

MIP Discovery, an innovative developer of non-biological affinity reagents designed to accelerate the development and production of cell and gene therapies, today announced it has closed a £7 million GBP (~$9 million USD) Series A financing round, led by Mercia Ventures.

21 Feb 2024

Unleashing the Potential of CRISPR-COPIES

The last ten years have seen tremendous advancements in CRISPR/Cas systems. These precise genome editing tools can be used for more than just gene therapy and transgenic crop development.

13 Feb 2024

Imec's Microfluidics Chip Shows Promise in Cancer Immune Profiling

The first clinical validation of Imec's cell sort chip technology has been reported in Cells through a new collaboration between Imec and the KU Leuven Laboratory of Tumor Immunology and Immunotherapy.

6 Feb 2024

Gene Therapy Can Interrupt the Progression of Deadly Inherited Heart Condition

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.

31 Jan 2024

Asimov Launches LV Edge Packaging System to Optimize Lentivirus Production

Asimov, the synthetic biology company advancing the design and manufacture of therapeutics, today announced the launch of their LV Edge Packaging System, which improves the cost efficiency and reduces the supply chain risk of lentiviral production.

30 Jan 2024

New Method Labels Viruses for Efficient Tracking and Tailoring

At the grocery store, barcodes make it quick and simple to identify products and frequently contain important details like quantity, location, and tracking.

22 Jan 2024

Phage Proteins Tame CRISPR-Cas3, Paving Way for Precise Gene Therapy

Through the use of CRISPR, an immune system that bacteria use to defend themselves against viruses, researchers have been able to alter genetic information within cells.

22 Jan 2024

Messenger RNA Therapy Could be Used to Correct Rare Liver Genetic Disease

By exploiting the technology used in Covid-19 vaccines, a team led by UCL, King's College London and Moderna scientists has created an effective therapy for a rare disease, in a study in mice, demonstrating the technology's potential therapeutic use in people.

16 Jan 2024

Breakthrough 3D Vascular Sheet Mimics Atherosclerosis for High-Throughput Drug Screening

A groundbreaking 3D, three-layer nanomatrix vascular sheet that possesses multiple features of atherosclerosis has been applied for developing a high-throughput functional assay of drug candidates to treat this disease, University of Alabama at Birmingham researchers report in the journal Biomaterials.

16 Jan 2024

Cracking the Code of the Cytoplasm: How Cells Organize Protein Production

Back in 2018, the lab of Christine Mayr, MD, PhD, at Memorial Sloan Kettering Cancer Center (MSK) introduced the world to a key cellular component that had been hiding in plain sight.

22 Dec 2023

Revealing the Molecular Machinery of Virus Warriors Against Bacterial Infections

The term “virus” is frequently linked with negative implications. Nevertheless, it is crucial to recognize that not all viruses are detrimental.

18 Dec 2023

Clean Cells Doubles Capacity with Opening of New Production Suites

Clean Cells today announces the launch of operations in its new production suites, located in the new facility opened in January 2023.

27 Nov 2023

Brunel University London Secures Japanese Patent for Gene Therapy Vector Safety Test

Gene therapy is a rapidly growing area of medical science. But there is little in the way of safety testing the vectors used to deliver gene therapy.

23 Nov 2023

Rare Genetic Variant Found to Protect Against Alzheimer's Disease

Researchers at Gladstone Institutes have discovered that a rare genetic variant known as the "Christchurch mutation" can block detrimental effects of apolipoprotein E4, the best-established risk factor for the most common form of Alzheimer's disease.

14 Nov 2023

Novel Gene Therapy Targets Core Motor Symptoms of Parkinson’s Disease

A team of researchers from the Shenzhen Institute of Advanced Technology (SIAT) at the Chinese Academy of Sciences (CAS) and their collaborators have devised a gene therapy approach aimed at selectively manipulating the affected circuitry in Parkinson’s disease.

6 Nov 2023

The Role of CRISPR-Cas9 in Treating Primary Immunodeficiency Disorders

Severe Combined Immunodeficiencies (SCIDs) encompass a collection of serious primary immunodeficiency disorders primarily instigated by genetic mutations that disrupt the development of T-cells.

30 Oct 2023