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.

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

New LMU-led DFG Research Unit eyes innovative gene therapies for retinal diseases

A new LMU-led DFG Research Unit is developing innovative approaches for treating retinal diseases.

15 Dec 2023

UCL researchers develop new gene therapy to cure severe childhood epilepsy

UCL researchers have developed a new gene therapy to cure a devastating form of childhood epilepsy, which a new study shows can significantly reduce seizures in mice.

14 Dec 2023

Gene therapy shows promise for arrhythmogenic cardiomyopathy

Researchers evaluated the effectiveness of adeno-associated virus (AAV)-mediated Plakophilin 2 (PKP2) delivery in treating arrhythmogenic cardiomyopathy (ACM).

12 Dec 2023

Small molecule applications of process Raman spectroscopy

In this interview, Kevin Broadbelt of Thermo Fisher Scientific discusses the small molecule applications of process Raman spectroscopy.

From Thermo Fisher Scientific – Portable and Handheld Raman Spectroscopy 11 Dec 2023

Study identifies targetable fusion RNAs in breast cancer

Comprehensive profiling of fusion RNAs present in a large cohort of metastatic breast tumors revealed unique fusion mutations that may be therapeutically targetable, according to results presented at the San Antonio Breast Cancer Symposium, held December 5-9, 2023.

8 Dec 2023

Study offers new hope for heart repair and regeneration with reduced mitochondrial activity

USF Health Heart Institute doctors are upbeat about cardiac regeneration. But when those batteries – heart muscle cells called cardiomyocytes − short circuit and die, the damage can be devastating.

8 Dec 2023

Many patients may not benefit from gene-editing treatments for sickle cell disease

Federal approval of a breakthrough gene-editing technology that treats the pain and debilitating effects of sickle cell disease is cause for celebration among a community with few options for relief.

8 Dec 2023

Liver trigger holds the key to boosting gene therapy success

Indiana University School of Medicine researchers have uncovered vital insights regarding a liver trigger that blocks an undesired immune response from gene therapy, surprisingly resulting in the activation of specific immune cells, despite the liver's typical role in suppressing immune responses.

7 Dec 2023

Novel mechanism leads to motor neuron degeneration in spinal muscular atrophy

The lab of Yongchao C. Ma, PhD, at Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA).

7 Dec 2023

Navigating STXBP1-related disorders: Unraveling clinical and developmental outcomes

Researchers examined the combined patient-years of seizure and developmental histories in individuals with disorders related to the STXBP1 gene.

4 Dec 2023

Texas Children's Hospital delivers first-ever gene therapy for Rett syndrome

Texas Children's Hospital, an internationally-recognized, top-ranked children's hospital and pediatric research center affiliated with Baylor College of Medicine, is the first to deliver a novel gene therapy to treat Rett syndrome in pediatric patients.

1 Dec 2023

CHOP researchers unlock 3D structure of crucial protein for cancer therapy

Chimeric Antigen Receptors (CARs) have opened up an exciting new field of therapeutic advancements for rare and difficult-to-treat cancers, as they have the ability to deliver targeted therapies that can kill tumor cells.

1 Dec 2023

Silencing galectin 1 protein shrinks liver cancer tumors in mice

Researchers at UC Davis Comprehensive Cancer Center have shown that inhibiting a specific protein using gene therapy can shrink hepatocellular carcinoma (HCC) in mice.

30 Nov 2023

Novel method can detect contaminants in T-cell cultures within 24 hours

Researchers from Critical Analytics for Manufacturing Personalized-Medicine (CAMP) Interdisciplinary Research Group (IRG) at Singapore-MIT Alliance for Research and Technology (SMART), MIT's research enterprise in Singapore, in collaboration with Singapore Centre for Environmental Life Sciences Engineering (SCELSE) and Massachusetts Institute of Technology (MIT), have developed a novel method capable of identifying contaminants in T-cell cultures within 24 hours.

27 Nov 2023

Synthetic biologists develop versatile platform for targeted drug delivery

In recent years, cell and gene therapies have shown significant promise for treating cancer, cystic fibrosis, diabetes, heart disease, HIV/AIDS and other difficult-to-treat diseases.

27 Nov 2023