Have You Heard? Viral-Mediated Gene Therapy Restores Hearing
نویسندگان
چکیده
Genetic loss of VGLUT3 in cochlear inner hair cells results in profound deafness. In this issue of Neuron, Akil et al. (2012) show that AAV-mediated introduction of wild-type VGLUT3 in the genetically deaf mouse cochlea results in significantly improved hearing.
منابع مشابه
Virally mediated Kcnq1 gene replacement therapy in the immature scala media restores hearing in a mouse model of human Jervell and Lange-Nielsen deafness syndrome
Mutations in the potassium channel subunit KCNQ1 cause the human severe congenital deafness Jervell and Lange-Nielsen (JLN) syndrome. We applied a gene therapy approach in a mouse model of JLN syndrome (Kcnq1(-/-) mice) to prevent the development of deafness in the adult stage. A modified adeno-associated virus construct carrying a Kcnq1 expression cassette was injected postnatally (P0-P2) into...
متن کاملBarriers and recent advances in non-viral vectors targeting the lungs for cystic fibrosis gene therapy
Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in CFTR genes that affect chloride ion channel. The CF is a good nominee for gene therapy as the asymptomatic carriers are phenotypically normal, and the desired cells are accessible for vector delivery. Gene therapy shows promising effects involving the correction of gene or replacement of the mutant gene with the func...
متن کاملKLF6 orchestrates cardiac myocyte-to-fibroblast communication: 'He who has ears to hear, let him hear'.
We try to help people listen to God’s Word in some practical ways. We print the Readings in the Bulletin so you can follow along if you wish; some people better concentrate on what is spoken when they have it written down in front of them. Or, if you have trouble hearing, the printed word is a great help. We have installed a public address system in church with microphones and speakers so the s...
متن کاملنگاهی به ژن درمانی، پیشرفتهای اخیر و چشم انداز آینده
Human gene therapy has attracted increasing attention as a highly encouraging therapeutic approach to treat wide variety of diseases, other than genetically inherited and monogenic disorders. This approach entails the introduction and expression of a variety of nucleic acids into human target cells for therapeutic purposes. In this article, we review the history, highlights, recently progresses...
متن کاملBeta thalassemia gene therapy using lentiviral vectors
Recent years, allogeneic bone marrow transplantation (BMT) has proved to be the successful cure for patients with thalassemia major, however this is restricted due to limited matched-related donor. Its complications include chronic graft-versus-host disease in 5-8% of patients. So, a molecular approach, such as gene therapy for direct normal beta globin gene transmission, seems quite promising ...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
عنوان ژورنال:
- Neuron
دوره 75 شماره
صفحات -
تاریخ انتشار 2012