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Bridging brain barriers for gene therapy
Reflecting on the challenges in treating brain diseases, this article explores ways to transduce the blood-brain barrier as well as the critical role of tanycytes as a target for gene therapy vectors.
Innovative gene therapy approaches for brain tumour-related epilepsy
Professor Mark Cunningham and Dr Kate Connor from Trinity College Dublin discuss the burden of brain tumour-related epilepsy and why novel therapies are urgently needed to improve the quality of life for those affected.
Development of a new gene therapy drug as a treatment for hypophosphatasia
The current primary treatment for hypophosphatasia is enzyme replacement therapy; however the development of a new gene therapy drug, ARU-2801, may change the landscape forever.
Exploring the science behind gene therapy in treating genetic diseases
Professor Alan Boyd explains the science behind gene therapy, the challenges in treating genetic diseases, and current trends in the sector.
Stem cell & gene therapy to treat osteogenesis imperfecta: hype or hope
Pascale V Guillot from University College London, Elizabeth Garrett Anderson Institute for Women’s Health, explains stem cell and gene therapy to treat osteogenesis imperfecta, but is this hype or hope?
What are the challenges surrounding gene therapy today?
Dr Jen Vanderhoven, Director, National Horizons Centre, Teesside University, sheds some light on how the gene therapy field can progress in the face of funding challenges and skills shortages.
Scientists successfully use gene therapy to restore eyesight
Scientists in France have partially restored the eyesight of a blind man, using gene therapy - he is now able to identify blurry shapes.
New gene therapy for eye disease developed in Ireland
Scientists from Trinity College Dublin have developed a new gene therapy for an eye disease that leads to progressive loss of vision.
Repurposing FDA-approved medicines and gene therapy to combat diabetic retinopathy
Lalit Singh Pukhrambam, PhD, and Ahmed S Ibrahim, PhD from Wayne State University School of Medicine in the U.S. focus here on the importance of combating diabetic retinopathy.
AAV: From almost a virus to an awesome vector for human gene therapy
Dr. Arun Srivastava, George H. Kitzman Professor of Genetics, from the University of Florida describes the rationale and strategies for the development of next generation (NextGen), generation X (GenX) and optimised (Opt) recombinant AAV vectors for human gene therapy
Recombinant AAV vectors have been, or are currently being, used in...
Changing the lives of people with hereditary angioedema through gene editing therapy
The lives of those who live with hereditary angioedema, a rare genetic disorder causing severe and unpredictable swelling attacks, may be transformed.
Eye health: Testing the safety of stem cell therapy for age-related macular degeneration
Kapil Bharti, PhD, Senior Investigator and Head of the Ocular and Stem Cell Translational Research Section at the National Eye Institute (NEI), explains testing the safety of a stem cell therapy for age-related macular degeneration in this special focus on eye health.
Duchenne muscular dystrophy: A test case for gene-targeted therapy development in rare disease
Laura Hagerty, PhD, Scientific Portfolio Director at the Muscular Dystrophy Association, highlights strategies for gene correction to treat Duchenne muscular dystrophy.
Nano DDS technology contributes to next-generation cancer immunotherapy
Dr Takashi Nakamura from Hokkaido University challenges the potential of nano-tech based drug delivery system (Nano DDS) in the development of next-generation cancer immunotherapy.
The regenerating spiny mouse and its potential for human therapy discovery
Discovering cures for human diseases or how to recover from damage or trauma to tissues is the fundamental goal of medical researchers, but it is a daunting task. Nevertheless, recent studies have shown the remarkable potential of the spiny mouse in addressing these crucial issues
Spinal muscular atrophy (SMA) – research from gene and modifiers to therapy
The fascinating research of Professor Brunhilde Wirth is placed under the spotlight, concerning her work in the field of disease-causing genes, modifier pathways and pathomechanisms of neuromuscular disorders, focussing on spinal muscular atrophy (SMA)
New concepts for the pathogenesis and asthma therapy
Prof Dr. Michael Roth, University Hospital Basel looks at how asthma therapy can reduce the effects of airway wall remodelling
Investigating lung cancer using genetically engineered mouse models (GEMMS)
Daniel J. Murphy, Professor of Lung Cancer & Mesothelioma at the University of Glasgow, School of Cancer Sciences, discusses opportunities for improving cancer research and care through the use of genetically engineered mouse models.
Knee OA therapy offers pain relief to patients
A patient with knee osteoarthritis (OA) provides a testimonial for "wonder" therapy that has relieved painful symptoms.
Internet-delivered cognitive behavioural therapy
Heather D. Hadjistavropoulos, Hugh C. McCall, and Jill A. B. Price, walk us through internet-delivered cognitive behavioral therapy tailored to public safety personnel.