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.

Nanoparticle-based gene therapy may hold promise for patients with asthma

A new study has demonstrated a way to deliver a nanoparticle-based gene therapy, in order to repair lungs damaged by chronic allergic asthma and to reduce inflammation that causes asthma attacks.

19 May 2016

CGT, University of Aberdeen announce creation of new company to develop novel technology for diabetes treatment

The Cell and Gene Therapy Catapult and University of Aberdeen, UK, today announce the creation of Islexa, a new company developing a novel technology to produce laboratory grown islets, the organoids responsible for insulin production.

18 May 2016

Researchers develop novel ss-siRNAs to turn off disease-related genes

Combining the therapeutic potential and advantages of existing oligonucleotide-based approaches to turn off disease-related genes, a type of single-stranded silencing RNAs (ss-siRNAs) has shown significantly improved potency and activity.

13 May 2016

Gene therapy shows promise in treating glioblastoma

A team from the International School for Advanced Studies in Trieste has obtained very promising results by applying gene therapy to glioblastoma.

13 May 2016

Researchers agree to test new stem cell gene therapy for Sanfilippo disease in human trial

Scientists in Manchester, who have developed a stem cell gene therapy to reverse a fatal childhood illness, have agreed to work with a new therapeutics company to test it in a human trial.

11 May 2016

Patient-specific immunotherapeutic vaccines may improve overall survival of melanoma patients

Two patients with melanoma that had spread to the liver survived for at least 8.5 and 12 years after resection of the hepatic tumor and treatment with patient-specific immunotherapeutic vaccines.

11 May 2016

Experimental therapy stops glioblastoma, high-grade gliomas in human cells and mouse models

Researchers report in the journal Cancer Cell an experimental therapy that in laboratory tests on human cells and mouse models stops aggressive, treatment-resistant and deadly brain cancers called glioblastoma and high-grade gliomas.

10 May 2016

Researchers identify new molecular targets for recovering motor function in men afflicted with SBMA

Michigan State University researchers used an old-fashioned neurobiology technique to explore new avenues for treatments to reverse a late-onset neurodegenerative disease that robs men of the capacity to walk, run, chew and swallow.

9 May 2016

Unique attributes of zebrafish may help study human blood disorders

Genetic regulation of the various types of blood cells in zebrafish and humans is highly similar, making it relatively easy and cost-effective to perform genetic, chemical, imaging and other molecular studies on this invaluable model organism to study normal hematopoetic development in humans as well as blood disorders and malignancies, as described in a Review article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

9 May 2016

Gene therapy could be potential treatment for neuropathic pain in cancer patients

A study providing new information about neuropathic pain afflicting some 90 percent of cancer patients who have had nerve damage caused by tumors, surgery, chemotherapy or radiation indicates gene therapy as a possible treatment.

6 May 2016

Globin gene transfer to treat beta-thalassemias shows promise in first clinical trial

Promising results from the first clinical trials of globin gene transfer to treat beta-thalassemias-inherited forms of anemia-have eliminated the need for blood transfusions in some individuals.

6 May 2016

Gene-replacement therapy could be potential treatment option for SMARD1

Spinal muscular atrophy (SMA) is a disease that causes progressive degeneration in the nerve cells that control muscles, thereby causing muscle weakness and eventually death.

6 May 2016

Researchers link global increase in type 1 diabetes to advances in medical care

Researchers from the University of Adelaide say the global increase in cases of type 1 diabetes is directly linked to advances in medical care, with the underlying genetics of the disease more likely to be passed from one generation to the next.

6 May 2016

Novel gene therapy can treat pulmonary hypertension linked with heart failure

Scientists have used a novel gene therapy to halt the progression of pulmonary hypertension, a form of high blood pressure in the lung blood vessels that is linked to heart failure, according to a study led by Roger J. Hajjar, MD, Professor of Medicine and Director of the Cardiovascular Research Center at the Icahn School of Medicine at Mount Sinai.

30 Apr 2016

Pioneering gene therapy may lead to potential cure for common causes of vision loss

Pioneering gene therapy has restored some vision to patients with a rare form of genetic blindness for as long as four years, raising hopes it could be used to cure common causes of vision loss, new University of Oxford research published today shows.

29 Apr 2016

New study on retroviral DNA could help improve treatments for HIV infection

When retroviruses such as HIV infect a cell, they first make a copy of their RNA genome in the form of DNA. The relatively short viral DNA strand then moves to the cell nucleus, where it inserts itself into the host cell's DNA.

27 Apr 2016

Modernization of screening methods in sperm banks could protect future children from highly heritable diseases

U.S. sperm banks perform genetic testing to screen for and disqualify carriers of a limited number of recessive disease mutations, but more comprehensive and affordable DNA-based screening methods are now available that can detect many more disease-causing genetic variations.

27 Apr 2016

Review focuses on autologous cell therapy that can be applied to cardiac surgery

The combination of cell and gene therapy is rapidly raising clinical interest, although their combination has been under investigation for several decades. This is mainly due to the availability and feasibility of clinically relevant gene delivery options. This review focuses on cell therapies for heart failure and their use combined with cardiac surgery as well as with gene therapy.

27 Apr 2016

Combination of nanoscale topography and triculture technology benefits large or slow-healing wounds

Large or slow-healing wounds that do not receive adequate blood flow could benefit from a novel approach that combines a nanoscale graft onto which three different cell types are layered. Proper cell alignment on the nanograft allows for the formation of new blood vessel-like structures, as reported in of Tissue Engineering, Part A, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free for download on the Tissue Engineering website until May 26, 2016.

27 Apr 2016

First breakthrough in gene therapy against aging

In September 2015, then 44 year-old CEO of BioViva USA Inc. Elizabeth Parrish received two of her own company's experimental gene therapies: one to protect against loss of muscle mass with age, another to battle stem cell depletion responsible for diverse age-related diseases and infirmities.

25 Apr 2016

Gene Therapy News and Research

Further Reading

Nanoparticle-based gene therapy may hold promise for patients with asthma

CGT, University of Aberdeen announce creation of new company to develop novel technology for diabetes treatment

Researchers develop novel ss-siRNAs to turn off disease-related genes

Gene therapy shows promise in treating glioblastoma

Researchers agree to test new stem cell gene therapy for Sanfilippo disease in human trial

Patient-specific immunotherapeutic vaccines may improve overall survival of melanoma patients

Experimental therapy stops glioblastoma, high-grade gliomas in human cells and mouse models

Researchers identify new molecular targets for recovering motor function in men afflicted with SBMA

Unique attributes of zebrafish may help study human blood disorders

Gene therapy could be potential treatment for neuropathic pain in cancer patients

Globin gene transfer to treat beta-thalassemias shows promise in first clinical trial

Gene-replacement therapy could be potential treatment option for SMARD1

Researchers link global increase in type 1 diabetes to advances in medical care

Novel gene therapy can treat pulmonary hypertension linked with heart failure

Pioneering gene therapy may lead to potential cure for common causes of vision loss

New study on retroviral DNA could help improve treatments for HIV infection

Modernization of screening methods in sperm banks could protect future children from highly heritable diseases

Review focuses on autologous cell therapy that can be applied to cardiac surgery

Combination of nanoscale topography and triculture technology benefits large or slow-healing wounds

First breakthrough in gene therapy against aging

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Automating research - solutions and best practices

How can microdialysis benefit drug development

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