In her third lecture, Zoghbi explores possible therapies for MECP2 disorders. First, using Deep Brain Stimulation (DBS), Zoghbi’s team together with collaborator Dr. Jianrong Tang were able to rescue learning and memory deficits, enhance neurogenesis, correct abnormal neural network activity, and improve MeCP2-linked gene expression changes in a mouse model of Rett Syndrome. Then, she discusses two approaches to normalize the MeCP2 protein in MECP2 duplication mice: by deleting the duplicated MECP2 gene in the genome, or by decreasing MECP2 mRNA levels using antisense-oligonucleotides. Even though these two approaches seem to rescue developmental issues caused by MECP2 duplication, titrating MeCP2 levels is required in order to avoid Rett-like symptoms caused by lowering the protein too much.
View the full talk with additional resources on our website
Rett Syndrome and the Insight it Provides into Syndromic Autism
Dr. Huda Zoghbi’s seminar takes us through the scientific journey from discovering the cause of Rett Syndrome to testing possible treatments for MECP2 disorders. (Talk recorded in December 2019)
- Part 1: Rett Syndrome: Genomes, Epigenomes and Neuropsychiatric conditionsAudience:
- Student
- Researcher
- Educators
- Educators of H. School / Intro Undergrad
- Educators of Adv. Undergrad / Grad
Duration: 00:33:43 - Part 2: Pathogenesis of MeCP2 DisordersAudience:
- Student
- Researcher
- Educators
- Educators of H. School / Intro Undergrad
- Educators of Adv. Undergrad / Grad
Duration: 00:26:12 - Part 3: Possible Future Therapies for Rett SyndromeAudience:
- Student
- Researcher
- Educators
- Educators of H. School / Intro Undergrad
- Educators of Adv. Undergrad / Grad
Duration: 00:27:32