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29 Jan Gene Therapy Restores Hearing In Trial of Autosomal Recessive Pediatric Deafness
Posted at 00:00h
in Author Interviews, Brigham & Women's - Harvard, Genetic Research, Hearing Loss, Lancet, Pediatrics
There has never been any treatment for any type hearing loss, including genetic hearing loss. This is the first time that a child born with complete hearing loss can regain hearing after gene therapy.
22 Mar Sickle Cell Anemia: Paying For Gene Therapy Presents Unique Challenges for Medicaid
While the price for any gene therapy may be impressive, the prevalence of the disease among health plan members is more consequential when considering affordability for a given payer.
08 Dec Gene Therapy Liberates Hemophilia B Patients from Requiring Regular Infusions of Clotting Factor
This looks to be a transformative therapy for patients and could be an option for patients across the adult lifespan - we treated patients from age 19 through 75.
01 Dec Gene Therapy for Mitochondrial Eye Disease Developed
Posted at 18:02h
in Author Interviews
We have shown that there is potential to use AAV delivered OPA1 based gene therapies for modulating cellular bioenergetics both in vitro and in vivo.
05 Apr Mouse Model Shows Gene Therapy Can Reverse Blue Cone Vision Disorder
MedicalResearch.com Interview with: Wen-Tao Deng, Ph.D. Department of Ophthalmology, College of Medicine| University of Florida, Gainesville, FL MedicalResearch.com: What is the background for this study? What are the main findings? Response: Blue cone monochromay (BCM) is a devastating vision disorder characterized by loss function of both L- and M-cones due to...15 Jan LUXTURNA Proves Effectiveness of Single Gene Therapy To Cure Rare Cause of Blindness
MedicalResearch.com Interview with: Dr. Stephen Rose PhD Chief Research Officer Foundation Fighting Blindness (FFB) Dr. Rose comments on the announcement of the FDA approval of voretigene neparvovec (LUXTURNA™) gene therapy for inherited blindness due to mutations in the RPE65 gene. What is the background for this announcement? What were the main findings...07 Jan Viral Vector Delivered Gene Therapy That Reversed Diabetes in Mice
Posted at 18:02h
in Annals Internal Medicine, Author Interviews, Diabetes, Genetic Research, University of Pittsburgh
MedicalResearch.com Interview with: Xiangwei Xiao, M.D., Ph.D. Assistant Professor of Department of Surgery, Children’s Hospital of Pittsburgh University of Pittsburgh School of Medicine, Pittsburgh, PA MedicalResearch.com: What is the background for this study? Response: Diabetes is a prevalent chronic disease characterized by persistently high blood glucose. Diabetes has two main...
07 Nov FDA Advisory Committee Recommended Approval of First Gene Therapy For Inherited Eye Disease
MedicalResearch.com Interview with: Dr. Stephen M. Rose, PhD Chief Research Officer Dr. Rose discusses the FDA advisory panel unanimously recommended approval of Spark Therapeutics’ Gene Therapy Luxturna for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal dystrophies, a group of rare blinding conditions caused by one...28 Aug Familial Hypercholesterolemia: “Junk” RNA May Facilitate Gene Therapy
MedicalResearch.com Interview with: Tamer Sallam, MD PhD Assistant Professor of Medicine Co-Director UCLA Center for Lipid Management Lauren B. Leichtman and Arthur E. Levine CDF Investigator Assistant Director, STAR Program Division of Cardiology, Department of Medicine David Geffen School of Medicine at UCLA Los Angeles, California 90095-1679 MedicalResearch.com: What is...17 Jul One Time Injection With Spark’s Gene Therapy LUXTURNA Demonstrated Lasting Visual Improvement
MedicalResearch.com Interview with: Stephen R. Russell, MD Dina J Schrage Professor of Macular Degeneration Research Service Director, Vitreoretinal Diseases and Surgery Professor of Ophthalmology and Visual Sciences The University of Iowa MedicalResearch.com: What is the background for this study? What are the main findings? Response: This study examines the efficacy...18 May Phase I Study Shows IV Gene Therapy May Improve Macular Degeneration
MedicalResearch.com Interview with: Prof Peter A Campochiaro MD Director, Retinal Cell and Molecular Laboratory Professor of Ophthalmology Johns Hopkins University School of Medicine Baltimore, MD MedicalResearch.com: What is the background for this study? What are the main findings? Response: Patients with wet age-related macular degeneration (AMD) have increased levels of...09 Feb Gene Therapy Restores Hearing Down To A Whisper, in Mice
Posted at 18:35h
in Author Interviews, Brigham & Women's - Harvard, Genetic Research, Hearing Loss, Nature
MedicalResearch.com Interview with: Gwenaelle Geleoc, PhD Assistant Professor Department of Otolaryngology F.M. Kirby Neurobiology Center Children’s Hospital and Harvard Medical School Boston, MA MedicalResearch.com: What is the background for this study? What are the main findings? Response: We seek to develop gene therapy to restore auditory and balance function in...
12 Oct Early Use of Gene Therapy May Stop Plaques of Alzheimer’s Disease
MedicalResearch.com Interview with: Dr. Magdalena Sastre PhD Faculty of Medicine, Department of Medicine Senior Lecturer Imperial College London MedicalResearch.com: What is the background for this study? Response: Alzheimer’s disease is the most common neurodegenerative disorder, affecting over 45 million people around the world. Currently, there are no therapies to cure...26 Aug Gene Therapy Delivers Intracellular Antibodies To Attack Abnormal Protein in Huntington’s Disease
MedicalResearch.com Interview with: Lee Henderson, Ph.D. CEO, Vybion, Inc. Ithaca, NY 14852 MedicalResearch.com: What is the background for this study? What are the main findings? Response: Huntington’s disease (HD) is a progressive and fatal neurodegenerative disease characterized by loss of both cognitive and motor function as a result of neuron loss...05 May Gene Therapy to Spinal Cord Offers Potential Hope to Peripheral Nerve Pain Patients
MedicalResearch.com Interview with: Hui-Lin Pan, MD, PhD Helen T. Hawkins Distinguished Professor and Deputy Division Head for Research Division of Anesthesiology and Critical Care, Unit 110 The University of Texas MD Anderson Cancer Center Houston, TX MedicalResearch.com: What is the background for this study? Dr. Hui-Lin Pan: Chronic nerve pain...04 Mar Gene Therapy Suggests Older Medication May Reduce Arrhythmias In Congenital Disorder
MedicalResearch.com Interview with: Silvia G Priori ,MD, PhD and Andrea Mazzanti, MD Medical Research: What is the background for this study? What are the main findings? Response: The study investigates a novel therapeutic approach for Long QT Syndrome type 3: a malignant varian of long QT Syndrome a disease in...22 Oct Gene Therapy with Leptin Induces Weight Loss
MedicalResearch.com Interview with: Urszula T. Iwaniec, Ph.D. Associate Professor Skeletal Biology Laboratory School of Biological and Population Health Sciences Oregon State University Corvallis, OR 97331 Medical Research: What is the background for this study? What are the main findings? Dr. Iwaniec: Excessive weight gain in adults is associated with a...12 Oct Promising Study Supports Gene Therapy For Wet Macular Degeneration
Posted at 12:23h
in Author Interviews, Genetic Research, Lancet, Macular Degeneration, Ophthalmology
MedicalResearch.com Interview with: Professor P. Elizabeth Rakoczy Centre for Ophthalmology and Visual Sciences The University of Western Australia Head of Department – Molecular Ophthalmology Lions Eye Institute Australia Medical Research: What is the background for this study? Prof. Rakoczy: Wet age related macular (wet-AMD) is the major cause of blindness in...
17 Jul Gene Therapy May Stop Blindness From Retinitis Pigmentosa
MedicalResearch.com Interview with: Professor Robert E MacLaren MB ChB DPhil FRCOphth FRCS Nuffield Laboratory of Ophthalmology Nuffield Department of Clinical Neurosciences Oxford Biomedical Research Centre, University of Oxford, Moorfields Eye Hospital & UCL NIHR Biomedical Research Centre for Ophthalmology London, UK. Medical Research: What is the background for this study?...13 Sep Study: Gene therapy causes breast cancer stem cells to self-destruct
Targeted agent avoids healthy cells, blocks proteins that prevent cell death in tumors HOUSTON — Gene therapy delivered directly to a particularly stubborn type of breast cancer cell causes the cells to self-destruct, lowers chance of recurrence and helps increase the effectiveness of some types of chemotherapy, researchers at The...
08 Jun Hebrew University: First WWOX Gene Replacement Therapy Administered to Child With Hereditary Seizures
For the first time, WWOX gene replacement therapy has been administered to a child with WOREE syndrome — a devastating epileptic encephalopathy. Here is the science behind the breakthrough and what it means for rare neurological diseases.
25 Jul Pimavanserin Therapy in Elderly Patients with Neurodegenerative Disease-Related Neuropsychiatric Symptoms : Safety Study
This lack of cognitive or motor function- related side effects further highlight the safety and tolerability of pimavanserin.
29 Mar One-Time Gene Replacement Therapy with Zolgensma Demonstrates Transformational Benefit For Spinal Muscular Atrophy Patients
MedicalResearch.com Interview with: Sitra Tauscher-Wisniewski, MD Vice President Clinical Development & Analytics Novartis Gene Therapies MedicalResearch.com: What is the background for this study? Would you briefly describe the condition of Spinal muscular atrophy (SMA)? Response: At the 2023 Muscular Dystrophy Association Conference, we presented new data from two of our...
16 Dec Gene Additive Therapy Prevents Pain Crisis Episodes in Sickle Cell Patients
The next big step is to achieve engraftment of genetically engineered stem cells without the need for the sickle cell patient to undergo conditioning treatment in the form of high dose chemotherapy.
03 Sep Adjuvant Therapy in Stage IIIA Melanoma: Cost vs Benefit of Gene Profiling
Posted at 09:47h
in Author Interviews, Cost of Health Care, Genetic Research, Immunotherapy, Melanoma, Surgical Research
How do patients value the trade-off between the up-front toxicity and cost of adjuvant therapy versus the reduced risk of recurrence?
14 May 21-Gene Recurrence Scores of 26 or Higher Can Help Determine If Chemotherapy Will Be Effective
Breast cancer patients with a 21-gene recurrence score of 26 or higher are a heterogeneous group of patients with different prognosis and outcomes.
08 May Early Breast Cancer: 21-Gene Recurrence Scores Can Help Guide Chemotherapy Prognosis
Breast cancer patients with a 21-gene recurrence score of 26 or higher are a heterogeneous group of patients with different prognosis and outcomes, and chemotherapy may benefit patients with recurrence scores of 26-30.
27 Feb Cancers That Survive Chemotherapy Acquire Resistance Genes in Different Ways
Each cancer that survived chemo harbored a different combination of mutations. However, mutations were significantly more common in genes that regulated cell proliferation.