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.

Researchers identify genetic switch that may be potential target for Alzheimer's disease

A team at the MRC Clinical Sciences Centre, based at Imperial College London, has found an important part of the machinery that switches on a gene known to protect against Alzheimer's Disease.

20 Sep 2016

New dermatological treatment could potentially help prevent or reverse diabetic neuropathy

For the 25 percent of type-2 diabetes patients who suffer from numbness and extreme nerve pain in their feet, a new dermatological treatment being tested by Northwestern Medicine scientists could potentially help prevent and maybe even reverse the neuropathy.

13 Sep 2016

Viral vector-based approaches could improve effectiveness of CFTR gene therapy

Cystic fibrosis (CF) is characterized by accumulation of thick mucus in the lungs and is associated with a high incidence of bacterial infection. Mutations in the gene encoding CF transmembrane conductance regulator (CFTR) underlie the disease.

9 Sep 2016

Penn scientists find striking similarities between human patients with LCA and dogs

Ciliopathies are diseases that affect the cilia, sensory organelles that most mammalian cells possess and which play a critical role in many biological functions.

30 Aug 2016

Kobe University researchers develop new technique for high-level genome editing operation

A team involving Kobe University researchers has succeeded in developing 'Target-AID', a genome editing technique that does not cleave the DNA.

26 Aug 2016

Former SpaceX engineers launch Synthego, CRISPRevolution to accelerate genome engineering research

Synthego, a leading provider of genome engineering solutions, officially launches today. Synthego’s flagship product line, CRISPRevolution, features synthetic guide RNA products designed to accelerate CRISPR/Cas9 and Cpf1 genome engineering research. CRISPRevolution is the first product line to offer economical access to fully synthetic RNA for high fidelity editing and increased precision in genome editing.

12 Aug 2016

ISCT selects London as host city for 2017 landmark conference to celebrate silver jubilee year

The International Society for Cellular Therapy, the global society of clinicians, researchers, regulatory specialists, technologists, and industry partners dedicated to the translation of cellular therapy into safe and effective therapies to improve patients’ lives, today announces they have awarded London as the host city for the landmark ISCT 2017 Annual Meeting to be held on May 3-6, at ExCeL London Exhibition and Convention Centre, UK to celebrate the Society’s 25th silver jubilee year. More than 1,600 international delegates are expected to attend.

10 Aug 2016

Researchers uncover how GWAS-implicated gene affects HDL-cholesterol levels

Researchers have uncovered how genes identified from genome-wide association studies (GWAS) affect high-density lipoprotein cholesterol (HDL-C), a biomarker of cardiovascular disease, after comparing several animal models with human patient data.

10 Aug 2016

Rice University bioengineer receives NIH grant to study use of AAV-based therapy to combat ovarian cancer

Rice University bioengineer Junghae Suh has been awarded a prestigious R01 grant by the National Institutes of Health to research the use of viral gene therapy to fight ovarian cancer.

3 Aug 2016

Next-generation sequencing to evaluate cell-free DNA yields more accurate results in diagnosis of MDS

Using next-generation sequencing (NGS) methods to analyze cell-free DNA in the blood of patients with myelodysplastic syndrome (MDS) yields more accurate results than the current standard approach of Sanger sequencing.

2 Aug 2016

Overcoming gene silencing barriers to target neurological conditions: an interview with Dr Errol de Souza

Many gene mutations that cause neurological disorders have been identified. For example, in a rare neurological disorder such as Huntington’s disease, an autosomal dominant mutation through expansion of CAG (cytosine-adenine-guanine) triplet repeats in the gene coding for the Huntingtin protein results in abnormal protein production.

1 Aug 2016

Triple-combination therapy patch shrinks tumors, prevents recurrence in colon cancer mice model

Investigators at Brigham and Women's Hospital have developed a hydrogel patch that can adhere to tumors in a preclinical model of colon cancer, delivering a local, combination treatment as the elastic gel breaks down over time.

29 Jul 2016

Study verifies human gene therapy in MPS I animal model

Researchers are closer to finding a better way to treat children with a rare metabolic disorder called MPS I. It is caused by a deficiency of the key enzyme IDUA needed to break down complex sugars in cells.

29 Jul 2016

New gene therapy shows promising results for treating neurodegenerative disorders

A new gene therapy approach designed to replace the enzyme that is deficient in patients with the inherited neurodegenerative disorders Tay-Sachs and Sandhoff diseases successfully delivered the therapeutic gene to the brains of treated mice, restored enzyme function, and extended survival by about 2.5-fold.

28 Jul 2016

Researchers test new approach to treat metabolic diseases without organ transplant

With a shortage of donor organs, Mayo Clinic is exploring therapeutic strategies for patients with debilitating liver diseases. Researchers are testing a new approach to correct metabolic disorders without a whole organ transplant. Their findings appear in Science Translational Medicine.

27 Jul 2016

Researchers find potential way for delivering gene therapy to treat eye diseases

Eye diseases such as diabetic retinopathy and age-related macular degeneration are among the leading causes of irreversible vision loss and blindness worldwide. Currently, gene therapy can be administered to treat these conditions -- but this requires an injection.

27 Jul 2016

Study reveals long-term safety of AAV2-neurturin gene therapy in patients with Parkinson's disease

New safety data from a study of patients with advanced Parkinson's disease five years after gene transfer-mediated delivery of the neuroprotective factor neurturin directly to patients' brains reveal no serious adverse events related to the treatment.

27 Jul 2016

Gene Therapy News and Research

Further Reading

Researchers identify genetic switch that may be potential target for Alzheimer's disease

New dermatological treatment could potentially help prevent or reverse diabetic neuropathy

Viral vector-based approaches could improve effectiveness of CFTR gene therapy

Penn scientists find striking similarities between human patients with LCA and dogs

Kobe University researchers develop new technique for high-level genome editing operation

Former SpaceX engineers launch Synthego, CRISPRevolution to accelerate genome engineering research

ISCT selects London as host city for 2017 landmark conference to celebrate silver jubilee year

Researchers uncover how GWAS-implicated gene affects HDL-cholesterol levels

Rice University bioengineer receives NIH grant to study use of AAV-based therapy to combat ovarian cancer

Next-generation sequencing to evaluate cell-free DNA yields more accurate results in diagnosis of MDS

Overcoming gene silencing barriers to target neurological conditions: an interview with Dr Errol de Souza

Triple-combination therapy patch shrinks tumors, prevents recurrence in colon cancer mice model

Study verifies human gene therapy in MPS I animal model

New gene therapy shows promising results for treating neurodegenerative disorders

Researchers test new approach to treat metabolic diseases without organ transplant

Researchers find potential way for delivering gene therapy to treat eye diseases

Study reveals long-term safety of AAV2-neurturin gene therapy in patients with Parkinson's disease

Researchers discover three new risk genes for ALS

Adhesive patch can deliver triple combination therapy to tumor sites

New virus-based method opens wide range of options to treat various diseases

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