Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies

Overview

Journal Front Cell Neurosci

Specialty Cell Biology

Date 2021 Jul 9

PMID 34239416

Citations 3

Authors

Georg von Jonquieres

Caroline D Rae

Gary D Housley

Affiliations

Soon will be listed here.

Abstract

Central Nervous System (CNS) homeostasis and function rely on intercellular synchronization of metabolic pathways. Developmental and neurochemical imbalances arising from mutations are frequently associated with devastating and often intractable neurological dysfunction. In the absence of pharmacological treatment options, but with knowledge of the genetic cause underlying the pathophysiology, gene therapy holds promise for disease control. Consideration of leukodystrophies provide a case in point; we review cell type - specific expression pattern of the disease - causing genes and reflect on genetic and cellular treatment approaches including hematopoietic stem cell gene therapies and approaches using adeno-associated virus (AAV) vectors. We link recent advances in vectorology to glial targeting directed towards gene therapies for specific leukodystrophies and related developmental or neurometabolic disorders affecting the CNS white matter and frame strategies for therapy development in future.

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