Genetics provides new individualized therapeutic targets for Parkinson’s disease

Garcia, Eric Joshua; Sidransky, Ellen

doi:10.4103/1673-5374.297076

Cited by 1 publication

(1 citation statement)

References 16 publications

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“…As with the other neurodegenerative diseases, there are both sporadic and genetic forms of PD ( Torrent et al, 2015 ). The death of dopaminergic neurons in familial PD is associated with oxidative stress and has been linked to several potentially pathogenic mutations in genes involved in mitochondrial homeostasis, such as PARK2 , PINK1 , PARK7 , SNCA , GBA1 , and LRRK2 ( Garcia and Sidransky, 2021 ). A large number of studies have used iPSC-derived iNs to study PD pathophysiology, with difficulties related to the epigenetic age of these cells (reviewed in Torrent et al, 2015 ).…”

Section: Applications For Induced Neurons Generated In Vitro Via Direct Neuronal Reprogrammingmentioning

confidence: 99%

Direct Neuronal Reprogramming: Bridging the Gap Between Basic Science and Clinical Application

Vasan

Park

David

et al. 2021

Front. Cell Dev. Biol.

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Direct neuronal reprogramming is an innovative new technology that involves the conversion of somatic cells to induced neurons (iNs) without passing through a pluripotent state. The capacity to make new neurons in the brain, which previously was not achievable, has created great excitement in the field as it has opened the door for the potential treatment of incurable neurodegenerative diseases and brain injuries such as stroke. These neurological disorders are associated with frank neuronal loss, and as new neurons are not made in most of the adult brain, treatment options are limited. Developmental biologists have paved the way for the field of direct neuronal reprogramming by identifying both intrinsic cues, primarily transcription factors (TFs) and miRNAs, and extrinsic cues, including growth factors and other signaling molecules, that induce neurogenesis and specify neuronal subtype identities in the embryonic brain. The striking observation that postmitotic, terminally differentiated somatic cells can be converted to iNs by mis-expression of TFs or miRNAs involved in neural lineage development, and/or by exposure to growth factors or small molecule cocktails that recapitulate the signaling environment of the developing brain, has opened the door to the rapid expansion of new neuronal reprogramming methodologies. Furthermore, the more recent applications of neuronal lineage conversion strategies that target resident glial cells in situ has expanded the clinical potential of direct neuronal reprogramming techniques. Herein, we present an overview of the history, accomplishments, and therapeutic potential of direct neuronal reprogramming as revealed over the last two decades.

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Section: Applications For Induced Neurons Generated In Vitro Via Direct Neuronal Reprogrammingmentioning

confidence: 99%

Direct Neuronal Reprogramming: Bridging the Gap Between Basic Science and Clinical Application

Vasan

Park

David

et al. 2021

Front. Cell Dev. Biol.

View full text Add to dashboard Cite

show abstract

Genetics provides new individualized therapeutic targets for Parkinson’s disease

Cited by 1 publication

References 16 publications

Direct Neuronal Reprogramming: Bridging the Gap Between Basic Science and Clinical Application

Direct Neuronal Reprogramming: Bridging the Gap Between Basic Science and Clinical Application

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