Gene editing holds the potential for a transformative approach to the therapy of CNS disorders. The emergence of novel gene-editing tools such as CRISPR, TALEN, Zinc Finger Nucleases and Homologous Recombination opens new possibilities in the development of disease relevant animal models and, more importantly, in highly specific therapeutic interventions.
In order for these technologies to come to fruition, several challenges need to be addressed, including efficient delivery to target areas and selective editing of neuronal and non-neuronal cell populations.
This workshop will introduce the potential applications of the gene-editing technology in rare neurodegenerative diseases from preclinical study design to therapeutic potential.
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Ottavio Vitolo, VP Clinical Development, Homology Medicines Inc
Whilst microglia have long been implicated in the pathology of neurodegenerative disease, recent accumulating evidence has pointed to these cell types as a driving factor in neuronal damage and disease progression for AD, PD and others.
Although different models have been established to study these diseases and cell type strategies, their effectiveness has been limited. In order to understand the interplay of underlying mechanisms and to investigate new therapeutic modalities, there is a need to increase human-relevance and accuracy. Recent technological advances, including IPSCs, now grant access to substantial quantities of disease-pertinent neurons, both with and without predisposing mutations.
Samuel Hasson, Principal Investigator, Neuroscience, Pfizer
Emerging data suggests that there is a link between changes in the gut microbiome and neurological diseases and disorders. This exciting bi-directional communication via the proposed gut-brain axis or GBA, has catapulted microbiota into the scientific spotlight for CNS disorders. However, with the size and complexity of the Gut-Brain Axis, there still remains a number of fundamental questions to allow scientific researchers to forge a successful path in demonstrating causality for a number of disease indications and ultimately developing target specific therapeutics.
As these two hot areas of research come together, now is the time to understand how we can continue to fuel this dynamic relationship.
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Patrice Garnier, CEO, Amabiotics