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Gene Medicine Xagena

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 ...



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