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.

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

Asimov launches LV Edge Packaging System to optimize lentivirus production

New off-the-shelf system reduces the cost and risk of lentiviral (LV) production for cell and gene therapy developers.

From Asimov 29 Jan 2024

New study of daylight vision using canine model offers critical insight into restoring cone function

In the retinas of human eyes, the cones are photoreceptor cells responsible for color vision, daylight vision, and the perception of small details.

27 Jan 2024

Prime editing takes gene therapy to the next level

This study is led by Prof. Xianqun Fan (Department of Ophthalmology, Shanghai Jiao Tong University School of Medicine Affiliated Ninth People's Hospital).

27 Jan 2024

Study shows effectiveness of gene therapy in treating congenital deafness

A new study co-led by investigators from Mass Eye and Ear, a member of Mass General Brigham, demonstrated the effectiveness of a gene therapy towards restoring hearing function for children suffering from hereditary deafness.

24 Jan 2024

Core Biogenesis and Nucleus Biologics announce strategic collaboration for manufacturing and distribution of cost effective and sustainable recombinant proteins

In a joint effort to provide lower cost and more defined reagents and media to the cell and gene therapy market, Nucleus Biologics, The Cell Performance Company™, a leading provider of custom cell culture media solutions for the cell and gene therapy industry, has signed a manufacturing, distribution, and collaboration agreement with Core Biogenesis, an innovator in the field of recombinant human proteins expressed in plants.

From Nucleus Biologics 24 Jan 2024

3D nanomatrix vascular sheet model for high-throughput atherosclerosis drug testing

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.

12 Jan 2024

mRNA therapy shows promise for curing children's rare liver 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.

10 Jan 2024

Marking a vital milestone for Vertex and Charles River

Charles River’s Memphis facility approved to manufacture Vertex’s CASGEVY™ therapy, the world’s first gene-edited therapy targeting severe sickle cell disease

From Charles River 9 Jan 2024

Cellular Origins, a TTP Company, establishes Advisory Board of global industry leaders

Renowned experts from across the CGT and robotics industries, Professor John Campbell, Dr Matthew Li, Dr Paul Roberts, and Geoffrey Hodge appointed to provide strategic and technical perspectives.

From Cellular Origins 9 Jan 2024

Revolutionary RNA-based switch offers new control over gene expression in mammalian cells

Researchers developed an RNA-based switch, the pA regulator system, to control gene expression in mammalian cells by modulating synthetic polyA signal cleavage, offering a novel approach for gene therapy applications.

4 Jan 2024

Small molecule-switchable RNA platform enables control of therapeutic protein expression

Just like a doctor adjusts the dose of a medication to the patient's needs, the expression of therapeutic genes, those modified in a person to treat or cure a disease via gene therapy, also needs to be maintained within a therapeutic window.

2 Jan 2024

Microbial decoration enhances drug delivery specificity and safety

Announcing a new publication for Acta Materia Medica journal. Microorganisms are mostly distributed on the surface of our skin and intestines and have crucial roles in physiologic and metabolic processes, such as digestion and immunity, which are closely related to diseases.

29 Dec 2023

Shedding new light on the hidden organization of the cytoplasm

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.

21 Dec 2023

Groundbreaking genome-editing technique holds promise for treating Charcot–Marie–Tooth disease

Scientific advances in the last century have changed our world significantly. For example, the world of genetics has opened doors to a myriad of possibilities: augmented human capabilities, cures for diseases, and even changes to the course of evolution.

19 Dec 2023

Gene Therapy News and Research

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