gene therapy - search results

A gene therapy has successfully restored hearing in five children with inherited deafness.

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.

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.

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.

Professor Alan Boyd explains the science behind gene therapy, the challenges in treating genetic diseases, and current trends in the sector.

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?

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 in France have partially restored the eyesight of a blind man, using gene therapy - he is now able to identify blurry shapes.

Scientists from Trinity College Dublin have developed a new gene therapy for an eye disease that leads to progressive loss of vision.

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.

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...

The lives of those who live with hereditary angioedema, a rare genetic disorder causing severe and unpredictable swelling attacks, may be transformed.

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.

Laura Hagerty, PhD, Scientific Portfolio Director at the Muscular Dystrophy Association, highlights strategies for gene correction to treat Duchenne muscular dystrophy.

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.

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

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)

Prof Dr. Michael Roth, University Hospital Basel looks at how asthma therapy can reduce the effects of airway wall remodelling

What if we could turn back the clock on age-associated dysfunctions by using a therapy that not only treats symptoms but acts to correct the underlying pathology and restores cells to normal function? Lori A. Birder and Edwin K. Jackson from the University of Pittsburgh School of Medicine, explain how this could be a possibility.

Associate Professor Ailsa McGregor from the University of Otago's School of Pharmacy has set on a mission to enhance the efficacy and safety of lithium treatment for patients battling bipolar disorder (BD).