ScienceDaily: Gene Therapy News

Discovery of a gene that controls three different diseases

Thu, 25 Apr 2013 13:26:26 EDT

Scientists have identified a single gene, ERCC4, that can be involved in three human diseases depending on which type of mutation it presents: Fanconi anaemia, xeroderma pigmentosum, or a type of progeria. The new findings add to existing knowledge of two DNA repair activities that are important in maintaining the stability of our genes and preventing cancer in the general population.

Repairing articular cartilage defects with an injectable gel engineered with gene modified BMSCs

Tue, 23 Apr 2013 14:43:43 EDT

Tissue engineering combined with gene therapy technology has the potential to manage the repair of defective articular cartilage. In this study, through minimally invasive injection methods the authors were able to repair rabbit articular cartilage defects with CS/PVA gel and gene modified BMSCs.

A noninvasive avenue for Parkinson's disease gene therapy

Sun, 21 Apr 2013 15:38:38 EDT

Researchers have developed a gene therapy approach that may one day stop Parkinson's disease in it tracks, preventing disease progression and reversing its symptoms. The novelty of the approach lies in the nasal route of administration and nanoparticles containing a gene capable of rescuing dying neurons in the brain.

Discovery of genetic defect which triggers epilepsy

Wed, 17 Apr 2013 09:19:19 EDT

Researchers have identified a gene behind an epilepsy syndrome, which could also play an important role in other idiopathic (genetically caused) epilepsies. With the so-called "next generation sequencing", with which genetic changes can be identified within a few days, it was ascertained that the CNTN2 gene is defective in this type of epilepsy.

One-two punch could be key in treating blindness

Tue, 09 Apr 2013 11:00:00 EDT

Researchers have discovered that using two kinds of therapy in tandem may be a knockout combo against inherited disorders that cause blindness. While their study focused on man's best friend, the treatment could help restore vision in people, too.

Lipid nanoparticles are ideal for delivering genes and drugs, researchers show

Thu, 28 Feb 2013 08:02:02 EST

Researchers are using nanotechnology to develop new formulations that can be applied to drugs and gene therapy. Specifically, they are using nanoparticles to design systems for delivering genes and drugs; this helps to get the genes and drugs to the point of action so that they can produce the desired effect.

Discovery in HIV may solve efficiency problems for gene therapy

Thu, 14 Feb 2013 13:26:26 EST

A research team has discovered an approach that could make gene therapy dramatically more effective for patients. The scientists discovered that the process of gene therapy is missing essential elements thereby reducing the effectiveness of this treatment. Re-introducing this element into their model system suggests that improvements for gene therapy areon the horizon.

Scientists prevent development of deafness in animals engineered to have Usher syndrome

Fri, 08 Feb 2013 15:28:28 EST

Most cases of congenital deafness are due to a mutation in a gene that is required for normal development of the sensory hair cells in the inner ear that are responsible for detecting sound. To cure deafness caused by such mutations, the expression of the gene must be corrected, a feat that has been elusive until recently.

Protein paves the way for correct stem cell differentiation

Thu, 07 Feb 2013 17:21:21 EST

A single embryonic stem cell can develop into more than 200 specialized cell types that make up our body. This maturation process is called differentiation and is tightly regulated. New research has identified a crucial role of the molecule Fbxl10 in differentiation of embryonic stem cells and suggests the molecule as a new potential target for cancer therapy.

Type 1 diabetes cured in dogs, study suggests

Thu, 07 Feb 2013 11:44:44 EST

Researchers have shown for the first time that it is possible to cure diabetes in large animals with a single session of gene therapy. After a single gene therapy session, the dogs recover their health and no longer show symptoms of the disease. In some cases, monitoring continued for over four years, with no recurrence of symptoms.

Gene may help predict best chemotherapy treatment for pancreatic cancer patients

Tue, 05 Feb 2013 12:34:34 EST

Researchers have identified a gene that may better predict survival for pancreatic adenocarcinoma, the fourth leading cause of cancer deaths in the United States. The scientists conducted a study that better defines the role of ribonucleotide reductase M1 (RRM1). The RRM1 gene encodes the regulatory subunit of ribonucleotide reductase, the molecular target of gemcitabine, a commonly used chemotherapy in pancreatic cancer.

Early-onset puberty in females explained

Tue, 29 Jan 2013 13:09:09 EST

This research provides significant insight into the reasons why early-onset puberty occurs in females. The researchers located key genes that trigger puberty and highlighted how external forces (epigenetics) appear to be a possible cause of early-onset puberty in some cases.

Gene mutation immortalizes malignant melanoma

Fri, 25 Jan 2013 10:42:42 EST

Scientists have discovered a previously unknown genetic cause of malignant melanoma: A gene mutation that leads to overactive telomerase, the so-called 'immortality enzyme.'

Light shed on complexity of gene therapy for congenital blindness

Mon, 21 Jan 2013 16:17:17 EST

Independent clinical trials have reported safety and efficacy for Leber congenital amaurosis (LCA), a congenital form of blindness caused by mutations in a gene (RPE65) required for recycling vitamin A in the retina. Now, new research finds that gene therapy for LCA shows enduring improvement in vision but also advancing degeneration of affected retinal cells, both in LCA patients and animal models of the same condition.

Quantum leap in gene therapy of Duchenne muscular dystrophy

Tue, 15 Jan 2013 11:17:17 EST

For years, scientists have been working to find the key to restoring dystrophin, but they have faced many challenges. After careful evaluation of 22 dogs, researchers found that the new version of the micro-dystrophin gene not only reduced inflammation and fibrosis, it also effectively improved muscle strength.

Researchers identify a new gene with a key role in obesity and diabetes

Thu, 10 Jan 2013 09:47:47 EST

An international team of scientists has identified a gene which regulates fat metabolism and is involved in the onset of obesity and related metabolic disorders like type 2 diabetes. The researchers regard this gene as a new therapeutic target for the treatment of obesity and insulin resistance in humans.

In epigenomics, location is everything: Researchers exploit gene position to test 'histone code'

Thu, 03 Jan 2013 13:07:07 EST

In a novel use of gene knockout technology, researchers tested the same gene inserted into 90 different locations in a yeast chromosome -- and discovered that while the inserted gene never altered its surrounding chromatin landscape, differences in that immediate landscape measurably affected gene activity.

Gene variation may shape bladder cancer treatment, study suggests

Thu, 03 Jan 2013 11:38:38 EST

Patients who have inherited a specific common genetic variant develop bladder cancer tumors that strongly express a protein known as prostate stem cell antigen (PSCA), which is also expressed in many pancreatic and prostate tumors, according to new research.

Rebuilding blood vessels through gene therapy

Fri, 21 Dec 2012 08:14:14 EST

Diagnosed with severe coronary artery disease, a group of patients too ill for or not responding to other treatment options decided to take part in a clinical trial testing angiogenic gene therapy to help rebuild their damaged blood vessels.

Serendipity points to new potential target and therapy for melanoma

Thu, 20 Dec 2012 14:39:39 EST

A new study describes a new target and potential treatment for melanoma, the most dangerous form of skin cancer. MicroRNA can decide which genes in a cell's DNA are expressed and which stay silent. Melanoma tends to lack microRNA-26a, which makes the gene SODD go silent.

First use of a gene therapy shows promise against fatal childhood disease

Wed, 19 Dec 2012 14:21:21 EST

There are promising results from the first-ever use of a virus-based gene therapy for a neurodegenerative/neurological disorder. The therapy was given to 19 young patients with Canavan disease, a devastating inherited childhood condition.

First gene therapy study in human salivary gland shows promise

Mon, 05 Nov 2012 15:13:13 EST

This finding comes from the first-ever Phase I clinical study of gene therapy in a human salivary gland. Its results show that the transferred gene, Aquaporin-1, has great potential to help head and neck cancer survivors who battle with chronic dry mouth. Aquaporin-1 encodes a protein that naturally forms pore-like water channels in the membranes of cells to help move fluid, such as occurs when salivary gland cells secrete saliva into the mouth.

Genetic predictors of fatigue for prostate cancer patients receiving androgen deprivation therapy

Fri, 26 Oct 2012 12:48:48 EDT

Researchers have found that men with prostate cancer who receive androgen deprivation therapy may predictably suffer from fatigue if they have single nucleotide polymorphisms in three pro-inflammatory genes. The discovery highlights the importance of personalized medicine, in which therapies are tailored to a patient’s genetic profile.

New anti-tumor cell therapy strategies are more effective

Thu, 25 Oct 2012 11:29:29 EDT

Targeted T-cells can seek out and destroy tumor cells that carry specific antigen markers. Two novel anti-tumor therapies that take advantage of this T-cell response are described in two new studies.

Gene polymorphisms identified that are responsible for breast density and cancer risk

Tue, 23 Oct 2012 20:46:46 EDT

It has long been known that breast density, or mammographic density, is a strong risk factor for breast cancer, and that estrogen and progestin hormone therapy increases dense breast tissue. Now, a study has identified several gene variants in hormone metabolism and growth factor pathways that may be associated with breast density and, hence, breast cancer risk.

New treatment method for children with brain tumours

Mon, 15 Oct 2012 10:09:09 EDT

Children who undergo brain radiation therapy run a significant risk of suffering from permanent neurocognitive adverse effects. These adverse effects are due to the fact that the radiation often encounters healthy tissue. This reduces the formation of new cells, particularly in the hippocampus – the part of the brain involved in memory and learning. Researchers in Sweden have used a model study to test newer radiation therapy techniques which could reduce these harmful adverse effects.

Shape matters in DNA nanoparticle therapy

Fri, 12 Oct 2012 10:21:21 EDT

Shape matters in DNA nanoparticle therapy: Particles could become a safer, more effective delivery vehicle for gene therapy

Fri, 12 Oct 2012 07:48:48 EDT

Researchers have discovered how to control the shape of nanoparticles that move DNA through the body and have shown that the shapes of these carriers may make a big difference in how well they work in treating cancer and other diseases. This study is also noteworthy because this gene therapy technique does not use a virus to carry DNA into cells.

Zinc fingers: A new tool in the fight against Huntington's disease

Wed, 10 Oct 2012 13:13:13 EDT

Huntington's disease (HD) is an inherited genetic disorder caused by the multiple repetition of a DNA sequence (the nucleotides CAG) in the gene encoding a protein called "Huntingtin". People who do not suffer from the disease have this sequence repeated 10 to 29 times. But in an affected person, the triplet is present more than 35 times. New research provides positive results reducing the chromosomal expression of the mutant gene, which would prevent the development of disease.

Gene therapy technique for children with immune disorder improved

Tue, 11 Sep 2012 12:53:53 EDT

Researchers have enhanced agene therapy approach for children with adenosine deaminase-deficient severe combined immunodeficiency using chemotherapy as conditioning regimen. For the first time, investigators test two different viral vectors to transport normal genes into the young patients' bone marrow stem cells and two different treatment plans in preparation for receiving gene therapy and compare outcomes.

Can gene therapy cure fatal diseases in children?

Wed, 05 Sep 2012 08:39:39 EDT

That low bone density causes osteoporosis and a risk of fracture is common knowledge. But an excessively high bone density is also harmful. The most serious form of excessively high bone density is a rare, hereditary disease which can lead to the patient’s death by the age of only five. Researchers in Sweden are now trying to develop gene therapy against this disease.

Promising new drug target discovered for treatment and prevention of heart failure

Sat, 25 Aug 2012 15:57:57 EDT

A promising new drug target for the treatment and prevention of heart failure has been discovered.

Elusive gene that causes a form of blindness from birth dlscovered

Sun, 29 Jul 2012 14:21:21 EDT

Researchers have isolated an elusive human gene that causes a common form of Leber congenital amaurosis (LCA), a relatively rare but devastating form of early-onset blindness. The new LCA gene is called NMNAT1. Finding the specific gene mutated in patients with LCA is the first step towards developing sight-saving gene therapy.

New gene mutation associated with congenital myopathy

Wed, 25 Jul 2012 14:25:25 EDT

Researchers have discovered a new cause of congenital myopathy: a mutation in a previously uncharacterized gene.

Gene therapy holds promise for reversing congenital hearing loss

Wed, 25 Jul 2012 13:22:22 EDT

A new gene therapy approach can reverse hearing loss caused by a genetic defect in a mouse model of congenital deafness, according to a preclinical study. The findings present a promising therapeutic avenue for potentially treating individuals who are born deaf.

New gene therapy strategy boosts levels of deficient protein in Friedreich’s ataxia

Wed, 25 Jul 2012 10:52:52 EDT

A novel approach to gene therapy that instructs a person’s own cells to produce more of a natural disease-fighting protein could offer a solution to treating many genetic disorders. The method was used to achieve a 2- to 3-fold increase in production of a protein deficient in patients with Friedreich’s ataxia.

Gene therapy treatment extends lives of mice with fatal disease

Mon, 16 Jul 2012 14:27:27 EDT

Scientists have found that introducing a missing gene into the central nervous system could help extend the lives of patients with Spinal Muscular Atrophy (SMA) -- the leading genetic cause of infantile death in the world.

Transforming cancer into a manageable illness with multi-drug approach

Wed, 11 Jul 2012 15:06:06 EDT

A new, multi-drug approach to treatment could make many cancers manageable, if not curable, illnesses by overcoming resistance to certain drug treatments, a new study suggests. The findings suggest that, of the billions of cancer cells that exist in a patient, only a tiny percentage -- about one in a million -- are resistant to drugs used in targeted therapy.

Melanoma-promoting gene discovered

Tue, 10 Jul 2012 09:34:34 EDT

Black skin cancer, also known as melanoma, is particularly aggressive and becoming increasingly common in Switzerland. Despite intensive research, however, there is still no treatment. Researchers have now discovered a gene that plays a central role in black skin cancer. Suppressing this gene in mice inhibits the development of melanoma and its proliferation – a discovery that could pave the way for new forms of therapy.

New gene transfer strategy shows promise for limb girdle and other muscular dystrophies

Mon, 09 Jul 2012 09:32:32 EDT

The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A new study describes a new gene therapy approach capable of delivering full-length versions of large genes and improving skeletal muscle function. The strategy may hold new hope for treating dysferlinopathies and other muscular dystrophies.

An economical, effective and biocompatible gene therapy strategy promotes cardiac repair

Fri, 06 Jul 2012 18:44:44 EDT

Medical researchers have established a novel hyperbranched poly(amidoamine) nanoparticle based hypoxia regulated vascular endothelial growth factor gene therapy strategy which is an excellent substitute for the current expensive and uncontrollable VEGF gene delivery system. This discovery provides an economical, feasible and biocompatible gene therapy strategy for cardiac repair.

Gene linked to facial, skull and cognitive impairment identified

Thu, 05 Jul 2012 17:20:20 EDT

A gene whose mutation results in malformed faces and skulls as well as mental retardation has been found by scientists.

Scientists develop alternative to gene therapy

Sun, 01 Jul 2012 19:16:16 EDT

Scientists have discovered a surprisingly simple and safe method to disrupt specific genes within cells. The scientists highlighted the medical potential of the new technique by demonstrating its use as a safer alternative to an experimental gene therapy against HIV infection.

How flu can cause severe infections

Thu, 28 Jun 2012 18:17:17 EDT

Scientists have discovered a new gene in the influenza virus that helps the virus control the body's response to infection. Although this control is exerted by the virus, surprisingly it reduces the impact of the infection.

Gold nanoparticles capable of 'unzipping' DNA

Wed, 20 Jun 2012 11:32:32 EDT

Gold nanoparticles with a slight positive charge work collectively to unravel DNA's double helix. This finding has ramifications for gene therapy research and the emerging field of DNA-based electronics.

New role for p53 genetic mutation -- initiation of prostate cancer

Thu, 07 Jun 2012 09:28:28 EDT

Researchers have found that a genetic mutation may play an important role in the development of prostate cancer. The mutation of the so-called p53 (or Tp53) gene was previously implicated in late disease progression, but until now has never been shown to act as an initiating factor. The findings may open new avenues for diagnosing and treating the disease.

Gene therapy can correct forms of severe combined immunodeficiency, study suggests

Thu, 24 May 2012 12:30:30 EDT

Scientists found that loss of the ADA gene directly contributes to B cell tolerance problems and that these defects are mostly corrected after gene therapy.

Children with rare, incurable brain disease improve after gene therapy

Wed, 16 May 2012 15:24:24 EDT

Taiwanese doctors have restored some movement in four children bedridden with a rare, life-threatening neurological disease using gene transfer. The first-in-humans achievement may also be helpful for more common diseases such as Parkinson's.

Light used to switch on gene expression

Thu, 10 May 2012 11:41:41 EDT

Imagine being able to control genetic expression by flipping a light switch. Researchers are using light-activated molecules to turn gene expression on and off. Their method enables greater precision when studying gene function, and could lead to targeted therapies for diseases like cancer.

Gene-modified stem cell transplant protects patients from toxic side effects of chemotherapy, study suggests

Wed, 09 May 2012 15:42:42 EDT

For the first time, scientists have transplanted brain cancer patients' own gene-modified blood stem cells in order to protect their bone marrow against the toxic side effects of chemotherapy. Initial results of the ongoing, small clinical trial of three patients with glioblastoma showed that two patients survived longer than predicted if they had not been given the transplants, and a third patient remains alive with no disease progression almost three years after treatment.

New gene thought to be cause in early-onset forms of Alzheimer's disease

Wed, 04 Apr 2012 10:29:29 EDT

A new gene that causes early onset of Alzheimer's disease has been discovered, researchers say. The research scientists showed that in the families of five of 14 patients suffering from the disease, mutations were detected on the gene SORL1.

Plant research reveals new role for gene silencing protein

Thu, 29 Mar 2012 14:19:19 EDT

A DICER protein, known to produce tiny RNAs in cells, also helps complete an important step in gene expression, according to research on Arabidopsis thaliana. The expression of a gene requires activation via a promoter or an external trigger. Plant research to be published in Science helps to show that later stages of transcription are just as important. This is likely to apply to other organisms, including humans.

World's first bedside genetic test

Thu, 29 Mar 2012 12:46:46 EDT

The world's first bedside genetic test has been developed. Scientists reports on the use of a simple cheek swab test, the Spartan RX CYP2C19, performed by nurses at the patient's bedside. This revolutionary technology allows doctors to rapidly identify patients with a genetic variant known as CYP2C19*2.

New gene therapy approach developed for red blood cell disorders

Tue, 27 Mar 2012 21:57:57 EDT

Scientists have designed what appears to be a powerful gene therapy strategy that can treat both beta-thalassemia disease and sickle cell anemia. They have also developed a test to predict patient response before treatment.

How a single gene mutation leads to uncontrolled obesity

Sun, 18 Mar 2012 14:39:39 EDT

Researchers have revealed how a mutation in a single gene is responsible for the inability of neurons to effectively pass along appetite suppressing signals from the body to the right place in the brain. What results is obesity caused by a voracious appetite.

Gene therapy for inherited blindness succeeds in patients' other eye

Wed, 08 Feb 2012 15:22:22 EST

Gene therapy for congenital blindness took another step forward, as researchers further improved vision in three adult patients previously treated in one eye. The patients were better able to see in dim light, with no adverse effects.

New RNA-based therapeutic strategies for controlling gene expression

Thu, 02 Feb 2012 16:48:48 EST

Small RNA-based nucleic acid drugs represent a promising new class of therapeutic agents for silencing abnormal or overactive disease-causing genes, and researchers have discovered new mechanisms by which RNA drugs can control gene activity.

Dyslexia-linked genetic variant decreases midline crossing of auditory pathways

Wed, 01 Feb 2012 09:29:29 EST

Scientists have found that a rare dyslexia-linked genetic variant of the ROBO1 gene decreases normal crossing of auditory pathways in the human brain. The results link, for the first time, a dyslexia-susceptibility gene to a specific sensory function of the human brain.

Gene therapy is a 'disruptive science' ready for commercial development, experts say

Tue, 24 Jan 2012 11:32:32 EST

The time for commercial development of gene therapy has come. Patients with diseases treatable and curable with gene therapy deserve access to the technology, which has demonstrated both its effectiveness and feasibility, says researchers.

Researchers develop gene therapy that could correct a common form of blindness

Mon, 23 Jan 2012 16:34:34 EST

A new gene therapy has the potential to treat a common form of blindness that strikes both youngsters and adults. The technique works by replacing a malfunctioning gene in the eye with a normal working copy that supplies a protein necessary for light-sensitive cells in the eye to function. Several complex steps remain before the gene therapy technique can be used in humans, but once at that stage, it has great potential to change lives.