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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...
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
Individualised Multimodal Immunotherapy beyond personalised medicine for brain cancer
Stefaan Van Gool, MD, PhD brings to light the important role of Individualised Multimodal Immunotherapy in improving the survival of brain cancer.
The future of living with Osteogenesis Imperfecta
CEO of Brittle Bone Society Patricia Osborne, revisits aspects of the condition and shares what is known around current medical research and treatments, speaking to experts attached to the Charity.
TheraVision: An oncolytic virus platform technology for a personalised virus-immunotherapy
Prof Dr Susanne M Bailer, Fraunhofer Institute for Interfacial Engineering and Biotechnology IGB, explains the possibilities of an oncolytic virus platform technology.
Exosome therapy for brittle bone disease
Pascale V Guillot, Associate Professor at University College London, investigates the possibility of exosome therapy for those living with brittle bone disease.
Prospects for minimally invasive and optimally effective cancer immunotherapy
Here, Professor of Biochemistry, Microbiology & Immunology at the University of Saskatchewan, Peter Betscher, explains how he is dedicated towards realising Robert North’s vision.
New gene-silencing drug could help with ALS
New research suggests that in suppressing a mutated ALS gene, it may be possible to delay to onset of symptoms.
Getting personal in brain tumour therapy: Matching therapies to patients
Sean E. Lawler PhD from Brown University Cancer Center, an experienced scientist in the brain tumour field
Genetic disease treatment research for creatine deficiency
Professor Olivier Braissant, believes there is hope for the treatment of creatine deficiency thanks to new genetic disease research - here, he tells us all about the recent developments.