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.

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

Novel Replacement Strategy Opens the Door to Therapeutic Interventions for Genomic Diseases

Severe Combined Immunodeficiencies (SCIDs) are a group of debilitating primary immunodeficiency disorders, primarily caused by genetic mutations that disrupt T-cell development.

27 Oct 2023

Novel Gene Therapy Approach Delivers Large Genes

A breakthrough by University of Zurich (UZH) researchers introduces a versatile technique for efficiently and safely delivering substantial genes, holding significant promise for therapeutic applications.

25 Oct 2023

Software Tool Paves the Way for Safer CRISPR Genome Editing

Researchers have developed a groundbreaking software tool known as DANGER analysis, offering a solution for safer and more precise genome editing.

23 Oct 2023

Effectiveness of SCID-X1 Gene Therapy Influenced by 3D Genome Architecture

Patients with X-linked severe combined immunodeficiency disorder (SCID-X1), often known as “bubble boy disease,” are born with a faulty gene that inhibits immune cells from being produced.

9 Oct 2023

Study Links Autism Gene to Broad Spectrum of Molecular and Cellular Defects

Researchers have used CRISPR gene editing, stem cells and human neurons to study the impact of a gene that is commonly mutated in autism.

6 Oct 2023

Improving the Efficiency of Gene Delivery to the Brain

Researchers from Rice University conducted tests to assess the safety and feasibility of gene delivery to various regions of the brain using a noninvasive technique based on ultrasound in rodents.

28 Sep 2023

Broken String Biosciences Closes $15M Series A Funding Round

Broken String Biosciences today announced that it has closed a $15 million Series A investment round, co-led by Illumina Ventures and Mérieux Equity Partners, with contributions from HERAN Partners, and existing investors Tencent and Dieter von Holtzbrinck Ventures.

19 Sep 2023

Unchained Labs Adds Big-Time Horsepower with New CCO

Unchained Labs, the life science tools company that’s all about getting biologics and gene therapy researchers the right tools for the job, announced today that life sciences ace, Scott Thomas, has joined the Company as Chief Commercial Officer.

From Unchained Labs 5 Sep 2023

New Gene Therapy Shows Promise in Treating Gliomas

The University of Michigan Department of Neurosurgery and Rogel Cancer Center study demonstrates enticing early results that a therapy merging cell-killing and immune-stimulating drugs is effective and safe in prolonging survival for patients with gliomas, an extremely aggressive form of brain cancer.

1 Sep 2023

Gene Therapy Shows Promise for Muscular Dystrophy Patients

Researchers at Cincinnati Children’s Hospital reveal how damage to the cell’s energy factory causes muscle wasting. Closing a pore in the mitochondrial membrane in gene-edited mice prevents disease progression.

28 Aug 2023

Waters Launches XBridge Premier GTx BEH SEC Columns for Gene Therapy Applications

Waters Corporation introduced the first in a new line of size exclusion chromatography (SEC) columns aimed at improving the analysis while lowering the cost of gene therapies, specifically adeno-associated viral (AAV) vectors.

From Waters Corporation 24 Aug 2023

PS Gene Editing Rebuilds Lost Neural Connections in Brain Disorder

A new study from the University of Minnesota is the first to show that gene therapy can mend neural connections in people with Hurler syndrome, a rare hereditary brain condition.

22 Aug 2023

Single Injection of CRISPR Gene-Editing Treatment Safely Removes SIV From Non-Human Primates

A single injection of a novel CRISPR gene-editing treatment safely and efficiently removes SIV – a virus related to the AIDS-causing agent HIV – from the genomes of non-human primates, scientists at the Lewis Katz School of Medicine at Temple University now report.

17 Aug 2023

Gene Therapy Prevents Progression of Kidney Disease in Children

Researchers at the University of Bristol have made a remarkable step forward in finding a potential cure for a type of childhood kidney disease.

11 Aug 2023

Cytomos Secures £4M to Advance New Approach to Cell Analysis

Cytomos, an Edinburgh-based life science company that has developed a proprietary new approach to analysing cells, has secured £4 million to scale up market-testing of its technology platform Cytomos Dielectric Spectroscopy (CDS).

9 Aug 2023

New Proof-of-Concept Model for Delivering Gene Editing Tools to Treat Blood Disorders

In a step forward in the development of genetic medicines, researchers at Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

28 Jul 2023

New Approach to Reverse Aging and Restore Cellular Function

Researchers have opened a new front in the battle against aging and diseases associated with old age through a ground-breaking study

13 Jul 2023

New Culture System may Improve Efficiency and Safety of Genome Editing in HSCs

Hematopoietic stem cells (HSCs) are rare cells found in the bone marrow that produce red blood cells, white blood cells, and platelets.

11 Jul 2023

Newly Found Disease Links Disruptions of Blood Formation, Immune System and Inflammation

In the quest to find the origin of the puzzling symptoms in four children, researchers from St. Anna Children's Cancer Research Institute, the CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences (ÖAW), and the Medical University of Vienna have discovered a completely new disease, linking disruptions of blood formation, the immune system, and inflammation.

22 Jun 2023