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Zheng-Yi Chen, D.Phil. Department of Otolaryngology-Head and Neck Surger Harvard Medical School Boston, MA

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.

Patrick DeMartino MD Pediatric Hematology and Oncology Fellow Doernbecher Children's Hospital Oregon Health & Science University

22 Mar Sickle Cell Anemia: Paying For Gene Therapy Presents Unique Challenges for Medicaid

Posted at 17:51h in Author Interviews, Cost of Health Care, Genetic Research, Hematology, JAMA

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.

Steven Pipe, MD Professor of Pediatrics and Pathology Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases Pediatric Medical Director, Hemophilia and Coagulation Disorders Program Director, Special Coagulation Laboratory University of Michigan

08 Dec Gene Therapy Liberates Hemophilia B Patients from Requiring Regular Infusions of Clotting Factor

Posted at 11:33h in Author Interviews, Genetic Research, Hematology

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

Posted at 18:54h in Author Interviews, Genetic Research, Ophthalmology

MedicalResearch.com Interview with: Wen-Tao Deng, Ph.D. Department of Ophthalmology, College of Medicine| University of Florida, Gainesville, FL MedicalResearch.com: What is...

15 Jan LUXTURNA Proves Effectiveness of Single Gene Therapy To Cure Rare Cause of Blindness

Posted at 23:38h in Author Interviews, FDA, Genetic Research, Ophthalmology

MedicalResearch.com Interview with: Dr. Stephen Rose PhD Chief Research Officer Foundation Fighting Blindness (FFB) Dr. Rose comments on the announcement...

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...

07 Nov FDA Advisory Committee Recommended Approval of First Gene Therapy For Inherited Eye Disease

Posted at 21:44h in Author Interviews, Genetic Research, Ophthalmology

MedicalResearch.com Interview with: Dr. Stephen M. Rose, PhD Chief Research Officer Foundation Fighting Blindness Dr. Rose discusses the FDA advisory panel...

28 Aug Familial Hypercholesterolemia: “Junk” RNA May Facilitate Gene Therapy

Posted at 21:44h in AHA Journals, Author Interviews, Genetic Research, Heart Disease, Lipids, UCLA

MedicalResearch.com Interview with: Tamer Sallam, MD PhD Assistant Professor of Medicine Co-Director UCLA Center for Lipid Management Lauren B. Leichtman...

17 Jul One Time Injection With Spark’s Gene Therapy LUXTURNA Demonstrated Lasting Visual Improvement

Posted at 22:13h in Author Interviews, Genetic Research, Lancet, Ophthalmology

MedicalResearch.com Interview with: Stephen R. Russell, MD Dina J Schrage Professor of Macular Degeneration Research Service Director, Vitreoretinal Diseases and...

18 May Phase I Study Shows IV Gene Therapy May Improve Macular Degeneration

Posted at 22:54h in Author Interviews, Lancet, Macular Degeneration, Ophthalmology

MedicalResearch.com Interview with: Prof Peter A Campochiaro MD Director, Retinal Cell and Molecular Laboratory Professor of Ophthalmology Johns Hopkins University...

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...

12 Oct Early Use of Gene Therapy May Stop Plaques of Alzheimer’s Disease

Posted at 11:17h in Alzheimer's - Dementia, Author Interviews, Genetic Research, PNAS

MedicalResearch.com Interview with: Dr. Magdalena Sastre PhD Faculty of Medicine, Department of Medicine Senior Lecturer Imperial College London MedicalResearch.com: What...

26 Aug Gene Therapy Delivers Intracellular Antibodies To Attack Abnormal Protein in Huntington’s Disease

Posted at 21:57h in Author Interviews, Genetic Research, Neurological Disorders

MedicalResearch.com Interview with: Lee Henderson, Ph.D. CEO, Vybion, Inc. Ithaca, NY 14852 MedicalResearch.com: What is the background for this study? What...

05 May Gene Therapy to Spinal Cord Offers Potential Hope to Peripheral Nerve Pain Patients

Posted at 21:01h in Author Interviews, Genetic Research, MD Anderson, Pain Research

MedicalResearch.com Interview with: Hui-Lin Pan, MD, PhD Helen T. Hawkins Distinguished Professor and Deputy Division Head for Research Division of...

04 Mar Gene Therapy Suggests Older Medication May Reduce Arrhythmias In Congenital Disorder

Posted at 13:52h in Author Interviews, Genetic Research, Heart Disease, JACC

MedicalResearch.com Interview with: Silvia G Priori ,MD, PhD and Andrea Mazzanti, MD Medical Research: What is the background for this...

22 Oct Gene Therapy with Leptin Induces Weight Loss

Posted at 17:46h in Author Interviews, Endocrinology, Genetic Research, Weight Research

MedicalResearch.com Interview with: Urszula T. Iwaniec, Ph.D. Associate Professor Skeletal Biology Laboratory School of Biological and Population Health Sciences Oregon...

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...

17 Jul Gene Therapy May Stop Blindness From Retinitis Pigmentosa

Posted at 14:33h in Author Interviews, Genetic Research, Ophthalmology

MedicalResearch.com Interview with: Professor Robert E MacLaren MB ChB DPhil FRCOphth FRCS Nuffield Laboratory of Ophthalmology Nuffield Department of Clinical...

13 Sep Study: Gene therapy causes breast cancer stem cells to self-destruct

Posted at 00:12h in Breast Cancer, MD Anderson

Targeted agent avoids healthy cells, blocks proteins that prevent cell death in tumors HOUSTON — Gene therapy delivered directly to...

Gus Alva, MD, DFAPA Medical Director, ATP Clinical Research Medical Director, Senior Brain Health, Hoag Hospital, Newport Beach, Assistant Professor, Department of Psychiatry and Neuroscience University of California, Riverside, CA

25 Jul Pimavanserin Therapy in Elderly Patients with Neurodegenerative Disease-Related Neuropsychiatric Symptoms : Safety Study

Posted at 11:35h in Author Interviews, Parkinson's

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

Posted at 13:47h in Author Interviews, Genetic Research, Neurological Disorders, Novartis

MedicalResearch.com Interview with: Sitra Tauscher-Wisniewski, MD Vice President Clinical Development & Analytics Novartis Gene Therapies MedicalResearch.com: What is the background...