GeneMedicine.net

Gene Medicine Xagena

Increased secretion of growth hormone leads to gigantism in children and acromegaly in adults; the genetic causes of gigantism and acromegaly are poorly understood. Researchers performed clinical a ...


Parkinson's disease is typically treated with oral dopamine replacement therapies; however, long-term treatment leads to motor complications and, occasionally, impulse control disorders caused by inte ...


Choroideremia is an X-linked recessive disease that leads to blindness due to mutations in the CHM gene, which encodes the Rab escort protein 1 ( REP1 ). The effects of retinal gene therapy with an a ...


Corneal graft rejection is a major problem in chronic herpetic keratitis (HK ) patients with latent infection. A new class of antiviral agents targeting latent and active forms of herpes simplex viru ...


National Institutes of Health ( NHI ) researchers have identified gene variants that cause a rare syndrome of sporadic fevers, skin rashes and recurring strokes, beginning early in childhood. The disc ...


The St. Jude Children's Research Hospital-Washington University Pediatric Cancer Genome Project has identified the most common genetic alteration ever reported in the brain tumor ependymoma and the ev ...


After a myocardial infarction, there is often permanent damage to a portion of the heart. This happens, in part, because cardiac muscle cells are terminally differentiated and cannot proliferate after ...


Researchers from the University of Washington and the HudsonAlpha Institute for Biotechnology have developed a new method for organizing and prioritizing genetic data. The Combined Annotation-Dependen ...


Nerve growth factor ( NGF ) is an endogenous neurotrophic-factor protein with the potential to restore function and to protect degenerating cholinergic neurons in Alzheimer's disease, but safe and eff ...


Choroideremia is an X-linked recessive disease that leads to blindness due to mutations in the CHM gene, which encodes the Rab escort protein 1 ( REP1 ). Researchers have assessed the effects of retin ...


Researchers have developed a novel strategy for treatment of Leber hereditary optic neuropathy ( LHON ) caused by a mutation in the nicotinamide adenine dinucleotide dehydrogenase subunit IV ( ND4 ) m ...


Successful gene therapy largely depends on the selective introduction of therapeutic genes into the appropriate target cancer cells. One of the most effective and promising approaches for targeting tu ...


Nonviral gene therapy represents a realistic option for clinical application in cancer treatment. A preclinical study has demonstrated the advantage of using the small-size MIDGE DNA vector for improv ...


Progranulin protein ( PGRN ) is a cysteine-rich growth factor encoded by the progranulin gene ( GRN ). PGRN mutations were identified in patients with frontotemporal lobar degeneration ( FTLD ) and re ...


Several epidemiological studies have investigated the associations of methylenetetrahydrofolate reductase ( MTHFR ) C677T and A1298C polymorphisms with hypertension ( H ) or hypertension in pregnancy ...


Despite the identification of gene mutations in methyl CpG binding protein 2 ( MECP2 ) being linked to Rett syndrome ( RS ), research has been hindered by the lack of commercially available reference ...


A new gene therapy for Parkinson's disease has achieved promising results in its first human tests, involving 15 patients.Nicholas Mazarakis, Gene Therapy at the Imperial College London, devised the a ...


Acute intermittent porphyria ( AIP ) is a rare genetic disease in which mutations in the porphobilinogen deaminase ( PBGD ) gene produce insufficient production of a protein necessary for heme synthes ...


Fanconi anemia ( FA ) is a complex genetic disease associated with congenital abnormalities, bone marrow failure ( BMF ) and cancer predisposition. Allogeneic transplantation constitutes the preferent ...


Researchers have previously reported the preliminary safety and efficacy data of a novel gene transfer approach in six subjects with severe haemophilia B ( Nathwani et al,NEJM365:2357–65, 2011 ). In b ...


Mucopolysaccharidosis type IIIA is a severe degenerative disease due to an autosomal recessive genetic defect in the gene coding for the lysosomal N-sulfoglycosamine sulfohydrolase ( SGSH ) whose cata ...


Pyruvate kinase deficiency ( PKD ) is a monogenic metabolic disease caused by mutations in the PKLR gene that impairs energetic balance in erythrocytes causing hemolytic anemia in a very variable rang ...


Whole-exome sequencing is a diagnostic approach for the identification of molecular defects in patients with suspected genetic disorders.Researchers have developed technical, bioinformatic, interpreti ...


Deep dermatophytosis is a severe and sometimes life-threatening fungal infection caused by dermatophytes. It is characterized by extensive dermal and subcutaneous tissue invasion and by frequent disse ...


Dyskeratosis congenita ( DC ) is a heterogeneous multi-system syndrome exhibiting marked clinical and genetic heterogeneity. In its classical form it is characterised by mucocutaneous abnormalities, b ...


The treatment of cancer has been one of the earliest and most frequent applications of gene therapy in experimental medicine. However, this indication entails unique difficulties, especially in the ca ...


Cellular immune responses to adeno-associated viral ( AAV ) vectors used for gene therapy have been linked to attenuated transgene expression and loss of efficacy. The impact of such cellular immune r ...


Transthyretin amyloidosis is caused by the deposition of hepatocyte-derived transthyretin amyloid in peripheral nerves and the heart. A therapeutic approach mediated by RNA interference ( RNAi ) could ...


Besides the use of Temozolomide ( Temodal ) and radiotherapy for patients with favourable methylation status, little progress has been made in the treatment of adult glioblastoma. Local control of the ...


Gene therapy of inherited diseases has provided convincing evidence of therapeutic benefits for many treated patients. In particular, treatment of primary severe congenital immunodeficiencies by gene ...


ProSavin is a gene-based treatment for Parkinson's disease, a chronic degenerative neurological condition. ProSavin uses LentiVector technology to deliver the genes for three enzymes that are required ...


Successful clinical response achieved in beta-thalassemia patient with single treatment of Bluebird Bio's lentiviral gene therapy Bluebird bio ( formerly Genetix Pharmaceuticals ), an emerging lead ...


All three people who received gene therapy at the University of Florida to treat a rare, incurable form of blindness have regained some of their vision. The report is published in the Human Gene Th ...


A study, published in the Nature Cancer Gene Therapy, has demonstrated that cancer cells in the liver are excellent targets for gene therapy using adenoviral vectors, based upon a fundamental new unde ...


New research suggests that gene therapy is a safe treatment method to explore in patients whose lower limbs are at risk for amputation because of poor circulation caused by blocked blood vessels. I ...


A novel gene therapy technique is safe and may be effective at staving off worsening symptoms of Parkinson's disease, according to the first scientific review of a dozen patients who have received the ...


Three young adults with an inherited form of blindness showed evidence of improved day and night vision following a specialized gene transfer procedure in a phase 1 clinical trial funded by the Nation ...


A cancer-suppressing gene has been successfully delivered into the tumors of stage 4 lung cancer patients via an intravenously administered lipid nanoparticle in a phase I clinical trial at The Univer ...


Combination gene therapy delivered in lipid-based nanoparticles drastically reduces the number and size of human non-small cell lung cancer tumors in mice. The study, by researchers at The University ...


Nearly one-third of American adults have high blood pressure, a major cause of myocardial infarctions, strokes and kidney failure. But a new technique tested at the University of Florida ( UF ) could ...


Researchers at the University of Pittsburgh School of Medicine have successfully protected mice against the damaging effects that radiation can have on bone marrow using gene therapy. Based on these r ...


Researchers at the University of Pittsburgh School of Medicine have used gene therapy to either completely abolish or significantly inhibit tumor progression in a mouse model of ovarian cancer. Th ...


Researchers at the University of Pittsburgh School of Medicine have successfully used gene therapy to accelerate muscle regeneration in experimental animals with muscle damage, suggesting this techniq ...


Reporting in the journal Genes and Development, investigators at The University of Texas M. D. Anderson Cancer Center have developed a simple way to use the RNAi approach to silence a selected gene in ...


Researchers at University of California, Berkeley, have shown that viruses can be forced to evolve in ways to benefit humans. The adeno-associated virus, or AAV, is a common, though innocuous, res ...


Reporting in the journal Genes and Development, investigators at The University of Texas M. D. Anderson Cancer Center have developed a simple way to use the RNAi approach to silence a selected gene in ...


One of the great challenges for treating Parkinson's diseases and other neurodegenerative disorders is getting medicine to the right place in the brain. The brain is a complex organ with many diff ...


Improved materials may allow stents to better deliver beneficial genes to patients with heart disease, by reducing the risk of inflammation that often negates initial benefits. The new technique, usi ...


Researchers at Cedars-Sinai Medical Center have developed a new method of signaling therapeutic genes to turn "off" or "on," a mechanism that could enable scientists to fine-tune genetic- and stem cel ...


In a study, published in the Nature Biotechnology, researchers led by a team of scientists at Memorial Sloan-Kettering Cancer Center have devised a novel strategy that uses stem cell-based gene therap ...



2000-2014© XAGENA srl - P.IVA: 04454930969 - REA: 1748680 - Tutti i diritti riservati