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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.
Gene therapy for LPLD patients linked to lower frequency and severity of pancreatitis

Gene therapy for LPLD patients linked to lower frequency and severity of pancreatitis

Up to 6 years after receiving a single treatment with the gene therapy product lipoprotein lipase (LPL), patients with the debilitating genetic disease LPL deficiency (LPLD) had about 50% fewer episodes of pancreatitis than before receiving the treatment. [More]
BREXIT vote threatens to disrupt advances in gene and cell therapies in the UK

BREXIT vote threatens to disrupt advances in gene and cell therapies in the UK

The recent BREXIT vote in the UK threatens to disrupt scientific progress and clinical advances in the development of innovative gene and cell therapies. [More]
Research shows degenerative diseases affecting the retina may be treatable with gene therapy

Research shows degenerative diseases affecting the retina may be treatable with gene therapy

Researchers have demonstrated the ability to deliver a fully functional copy of the CLN3 gene to stem cells of patients with juvenile NCL, an inherited neurodegenerative disease in which a mutation in the CLN3 gene causes early-onset severe central vision loss. [More]
Gene editing of hematopoietic stem cells can cure many hereditary and congenital diseases

Gene editing of hematopoietic stem cells can cure many hereditary and congenital diseases

Recent advances in gene editing technology, which allows for targeted repair of disease-causing mutations, can be applied to hematopoietic stem cells with the potential to cure a variety of hereditary and congenital diseases. [More]
Color-deficiency or color-blindness?

Color-deficiency or color-blindness?

Color blindness could be considered a bit of a misleading term, because there are very few people who can't see color at all. Most people have what we call color deficiency or color confusion, which means that they're not blind to color; they just see a reduced number of colors. [More]
Scientists engineer smallest-reported synthetic virus that may help advance gene therapy

Scientists engineer smallest-reported synthetic virus that may help advance gene therapy

Gene therapy is a kind of experimental treatment that is designed to fix faulty genetic material and help a patient fight off or recover from a disease. [More]
Lund University stem cell researcher awarded Fernström prize for study on repairing damaged brain

Lund University stem cell researcher awarded Fernström prize for study on repairing damaged brain

Is it possible to convert a patient’s own skin cells into functioning nerve cells? Or insert healthy genes to reprogram the cells of a damaged brain? Stem cell researcher Malin Parmar at Lund University in Sweden is studying these types of issues, in close collaboration with clinical researchers. [More]
Gene therapy may be viable approach for treating CF lung problems

Gene therapy may be viable approach for treating CF lung problems

Two new studies from the University of Iowa suggest that gene therapy may be a viable approach for treating or preventing lung disease caused by cystic fibrosis. [More]
Researchers identify genetic switch that may be potential target for Alzheimer's disease

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. [More]
New dermatological treatment could potentially help prevent or reverse diabetic neuropathy

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. [More]

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. [More]
Penn scientists find striking similarities between human patients with LCA and dogs

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. [More]
Kobe University researchers develop new technique for high-level genome editing operation

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. [More]
Former SpaceX engineers launch Synthego, CRISPRevolution to accelerate genome engineering research

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. [More]
ISCT selects London as host city for 2017 landmark conference to celebrate silver jubilee year

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. [More]
Researchers uncover how GWAS-implicated gene affects HDL-cholesterol levels

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

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

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

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. [More]
Triple-combination therapy patch shrinks tumors, prevents recurrence in colon cancer mice model

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. [More]
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