Publications and Presentations
Direct Image Guided Convective Perfusion of the Bilateral Thalami Offers a Consistent Approach to CNS Dosing: First-in-Human Experience with Gene Therapy for Frontotemporal Dementia
Date: May 16, 2025
Conference / Journal: ASCGT 2025
Author(s): D.L.Cooper
Effective knockdown of ATXN2 following intracerebroventricular administration of AVB-205 construct in BAC-ATXN2-Q72 transgenic mice
Date: Dec 7, 2024
Conference / Journal: International Symposium on ALS/MND
Author(s): P.M. Coan
A Translational Assay for Ataxin-2 Related Drug Development Products
Date: Dec 7, 2024
Conference / Journal: International Symposium on ALS/MND
Author(s): B. Rougemont
Effective SOD1 targeting with vMiX™, an innovative AAV-based RNA interference platform
Date: Dec 7, 2024
Conference / Journal: International Symposium on ALS/MND
Author(s): L. Reilly
vMiX , an innovative AAV-based RNA interference platform: from in vitro development to in vivo validation by targeting genes involved in ALS
Date: Oct 24, 2024
Conference / Journal: ESGCT 2024
Author(s): R. Joubert
Effective SOD1 targeting with vMiXTM, an innovative AAV-based RNA interference platform
Date: Oct 23, 2024
Conference / Journal: ESGCT 2024
Author(s): L. Reilly
Development of an in vitro potency assay for AVB-101, a recombinant AAV9 gene therapy for the treatment of frontotemporal dementia with progranulin mutations
Date: Oct 23, 2024
Conference / Journal: ESGCT 2024
Author(s): R. Lyth
Development of a triplex one-step RT-ddPCR method as a quantitative potency assay for the vMiXTM platform
Date: Oct 23, 2024
Conference / Journal: ESGCT 2024
Author(s): R. Lyth
The vMiX™ platform: Tailored miRNA gene therapy targeting molecular drivers of ALS and FTD
Date: Oct 22, 2024
Conference / Journal: ESGCT 2024
Author(s): A. Ule
Effective knockdown of ATXN2 following intracerebroventricular administration of AVB-205 construct in BAC-ATXN2-Q72 transgenic mice
Date: Oct 22, 2024
Conference / Journal: ESGCT 2024
Author(s): P.M. Coan
Development of HPLC-based methods for the systematic quantification of multiple quality attributes of AAV9 preparations
Date: Oct 22, 2024
Conference / Journal: ESGCT 2024
Author(s): A. Martorana
Pre-Clinical Development of AVB-101, an AAV Gene Therapy Treatment for Frontotemporal Dementia with Progranulin Mutations
Date: Sep 21, 2024
Conference / Journal: ISFTD 2024
Author(s): Youn Bok Lee PhD
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