Author Interviews, Brigham & Women's - Harvard, Genetic Research, Hearing Loss, Lancet, Pediatrics / 29.01.2024
Gene Therapy Restores Hearing In Trial of Autosomal Recessive Pediatric Deafness
MedicalResearch.com Interview with:
[caption id="attachment_61287" align="alignleft" width="180"]
Dr. Zheng-Yi Chen[/caption]
Zheng-Yi Chen, D.Phil.
Department of Otolaryngology-Head and Neck Surger
Harvard Medical School
Boston, MA
MedicalResearch.com: What is the background for this study? Would you briefly explain the process and indication
Response: This clinical trial is to use gene therapy to treat a type of genetic hearing loss. Genetic hearing loss mainly affects children. One in 600 newborns can have genetic hearing loss. There is no drug treatment for any type of hearing loss except for cochlear implants, which have limitations. This study focuses on a type of genetic hearing loss, DFNB9, due to a missing gene called Otoferlin. Without Otoferlin, children are born with complete hearing loss and without the capacity to speak. The goal of the trial is to study if gene therapy is safe and efficacious in treating children so they can regain hearing and the ability to speak.
Dr. Zheng-Yi Chen[/caption]
Zheng-Yi Chen, D.Phil.
Department of Otolaryngology-Head and Neck Surger
Harvard Medical School
Boston, MA
MedicalResearch.com: What is the background for this study? Would you briefly explain the process and indication
Response: This clinical trial is to use gene therapy to treat a type of genetic hearing loss. Genetic hearing loss mainly affects children. One in 600 newborns can have genetic hearing loss. There is no drug treatment for any type of hearing loss except for cochlear implants, which have limitations. This study focuses on a type of genetic hearing loss, DFNB9, due to a missing gene called Otoferlin. Without Otoferlin, children are born with complete hearing loss and without the capacity to speak. The goal of the trial is to study if gene therapy is safe and efficacious in treating children so they can regain hearing and the ability to speak.
Dr. Tian[/caption]
MedicalResearch.com Interview with:
Maoyi TIAN PhD
Program Head, Digital Health and Head, Injury & Trauma
Senior Research Fellow
The George Institute
MedicalResearch.com: What is the background for this study?
Response: There is clear evidence from the literature that sodium reduction or potassium supplementation can reduce blood pressure. Reduced blood pressure can also lead to a risk reduction for cardiovascular diseases.
Salt substitute is a reduced sodium added potassium product combined those effects. Previous research of salt substitute focus on the blood pressure outcome. There is no evidence if salt substitute can reduce the risk of cardiovascular diseases or pre-mature death. This study provided a definitive evidence for this unaddressed question.
MedicalResearch.com: What are the main findings?
The main findings of the research were: