PMID- 33802760
OWN - NLM
STAT- MEDLINE
DCOM- 20210422
LR  - 20211204
IS  - 1422-0067 (Electronic)
IS  - 1422-0067 (Linking)
VI  - 22
IP  - 6
DP  - 2021 Mar 17
TI  - Therapeutic Potential of AAV1-Rheb(S16H) Transduction against Neurodegenerative 
      Diseases.
LID - 10.3390/ijms22063064 [doi]
LID - 3064
AB  - Neurotrophic factors (NTFs) are essential for cell growth, survival, synaptic 
      plasticity, and maintenance of specific neuronal population in the central 
      nervous system. Multiple studies have demonstrated that alterations in the levels 
      and activities of NTFs are related to the pathology and symptoms of 
      neurodegenerative disorders, such as Parkinson's disease (PD), Alzheimer's 
      disease (AD), and Huntington's disease. Hence, the key molecule that can regulate 
      the expression of NTFs is an important target for gene therapy coupling 
      adeno-associated virus vector (AAV) gene. We have previously reported that the 
      Ras homolog protein enriched in brain (Rheb)-mammalian target of rapamycin 
      complex 1 (mTORC1) axis plays a vital role in preventing neuronal death in the 
      brain of AD and PD patients. AAV transduction using a constitutively active form 
      of Rheb exerts a neuroprotective effect through the upregulation of NTFs, thereby 
      promoting the neurotrophic interaction between astrocytes and neurons in AD 
      conditions. These findings suggest the role of Rheb as an important regulator of 
      the regulatory system of NTFs to treat neurodegenerative diseases. In this 
      review, we present an overview of the role of Rheb in neurodegenerative diseases 
      and summarize the therapeutic potential of AAV serotype 1 (AAV1)-Rheb(S16H) 
      transduction in the treatment of neurodegenerative disorders, focusing on 
      diseases, such as AD and PD.
FAU - Nam, Youngpyo
AU  - Nam Y
AD  - Brain Science and Engineering Institute, Kyungpook National University, Daegu 
      41944, Korea.
FAU - Moon, Gyeong Joon
AU  - Moon GJ
AUID- ORCID: 0000-0002-2851-9563
AD  - Center for Cell Therapy, Asan Institute for Life Science, Asan Medical Center, 
      Seoul 05505, Korea.
AD  - Department of Convergence Medicine, University of Ulsan College of Medicine, 
      Seoul 05505, Korea.
FAU - Kim, Sang Ryong
AU  - Kim SR
AUID- ORCID: 0000-0003-0299-1613
AD  - Brain Science and Engineering Institute, Kyungpook National University, Daegu 
      41944, Korea.
AD  - School of Life Sciences, Kyungpook National University, Daegu 41566, Korea.
AD  - BK21 FOUR KNU Creative BioResearch Group, Kyungpook National University, Daegu 
      41566, Korea.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20210317
PL  - Switzerland
TA  - Int J Mol Sci
JT  - International journal of molecular sciences
JID - 101092791
RN  - 0 (Nerve Growth Factors)
RN  - 0 (Ras Homolog Enriched in Brain Protein)
RN  - Adeno-associated virus-1
SB  - IM
MH  - Animals
MH  - Dependovirus
MH  - Humans
MH  - Models, Biological
MH  - Nerve Growth Factors/metabolism
MH  - Neurodegenerative Diseases/*therapy
MH  - Parvovirinae/*metabolism
MH  - Ras Homolog Enriched in Brain Protein/*metabolism
MH  - *Transduction, Genetic
PMC - PMC8002454
OTO - NOTNLM
OT  - Alzheimer's disease
OT  - Parkinson's disease
OT  - Rheb(S16H)
OT  - neurodegenerative disease
OT  - neurotrophic factor
COIS- The authors declare that there is no conflict of interest.
EDAT- 2021/04/04 06:00
MHDA- 2021/04/23 06:00
PMCR- 2021/03/17
CRDT- 2021/04/03 01:12
PHST- 2021/01/25 00:00 [received]
PHST- 2021/03/08 00:00 [revised]
PHST- 2021/03/16 00:00 [accepted]
PHST- 2021/04/03 01:12 [entrez]
PHST- 2021/04/04 06:00 [pubmed]
PHST- 2021/04/23 06:00 [medline]
PHST- 2021/03/17 00:00 [pmc-release]
AID - ijms22063064 [pii]
AID - ijms-22-03064 [pii]
AID - 10.3390/ijms22063064 [doi]
PST - epublish
SO  - Int J Mol Sci. 2021 Mar 17;22(6):3064. doi: 10.3390/ijms22063064.