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.

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

Gene therapy could reverse symptoms of rare, hereditary DOOR syndrome

A lot of research has been done over many decades on diseases that are widespread in large parts of the population, such as cancer and heart disease. As a result, treatment methods have improved enormously thanks to long-term research efforts on diseases that affect many people.

23 Nov 2023

Genetic vulnerability of leukemia cells uncovered

Leukemia is the most common type of cancer in children. Treatment involves intensive chemotherapy, which has severe side effects due to its non-specific mode of action.

22 Nov 2023

Combined application of therapeutic viruses and nanomaterials to revolutionize cancer therapy

Using oncolytic viruses (OVs) in combination with nanomaterials as a novel approach to cancer treatment.

22 Nov 2023

Long-term monitoring of gene therapy patients crucial for safety, study says

Medical research has shown promising results regarding the potential of gene therapy to cure genetic conditions such as sickle cell disease and the findings of this study, published in Nature Medicine, offer important new insights into processes happening in the body after treatment.

16 Nov 2023

Gene therapy for rare diseases: A decade of progress and remaining challenges

A new review article in the peer-reviewed journal Human Gene Therapy summarizes the significant milestones in the development of gene therapy medicinal products that have facilitated the treatment of a significant number of rare diseases.

14 Nov 2023

"Christchurch mutation" can block detrimental effects of Alzheimer’s risk factor

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.

13 Nov 2023

Unlocking the cure for hepatitis B virus: Challenges and opportunities

Hepatitis B virus (HBV) is a significant public health problem, with over 296 million people chronically infected worldwide.

13 Nov 2023

Gene therapy's double-edged sword: Breakthrough treatments face manufacturing and efficacy

Research explores the advancements, challenges, and economic implications of gene therapy for various diseases, including inherited blood disorders, malignancies treated with CAR-T cells, and diseases targeted by in vivo AAV vectors. The review highlights the transformative potential of gene therapies while acknowledging the complexities in manufacturing, safety, efficacy, and long-term durability.

12 Nov 2023