PMID- 29856034
OWN - NLM
STAT- MEDLINE
DCOM- 20190218
LR  - 20190219
IS  - 1940-6029 (Electronic)
IS  - 1064-3745 (Linking)
VI  - 1780
DP  - 2018
TI  - Disease Modification Through Trophic Factor Delivery.
PG  - 525-547
LID - 10.1007/978-1-4939-7825-0_24 [doi]
AB  - Huntington's disease (HD) is characterized by a significant loss of striatal 
      neurons that project to the globus pallidus and substantia nigra, together with 
      loss of cortical projection neurons in varying regions. Mutant huntingtin is 
      suggested to drive the pathogenesis partially by downregulating corticostriatal 
      brain-derived neurotrophic factor (BDNF) levels and signaling. Neurotrophic 
      factors are endogenous peptides that promote the survival and maintenance of 
      neurons. BDNF and other neurotrophic factors have shown neuroprotective benefits 
      in various animal models of neurodegeneration, and are interesting candidates to 
      protect the cell populations that are destined to die in HD. In an attempt to 
      enhance the delivery of neurotrophic factors, several methods have been 
      established to deliver long-term neurotrophic factor gene therapy to human target 
      tissues. This chapter discusses two alternative approaches that have been shown 
      to have potential to deliver neurotrophic factors as a neuroprotective gene 
      therapy for HD. The methods are (1) ex vivo approach where encapsulated cells 
      engineered to express neurotrophic factor are inserted into brain parenchyma or 
      ventricle, and (2) in vivo viral vector therapy, in which viral vector is 
      injected into desired brain area to express gene of interest in the host cells.
FAU - Savolainen, Mari
AU  - Savolainen M
AD  - Department of Neurological Sciences, Rush University Medical Center, Chicago, IL, 
      USA.
FAU - Emerich, Dwaine
AU  - Emerich D
AD  - NsGene, Inc., Providence, RI, USA.
FAU - Kordower, Jeffrey H
AU  - Kordower JH
AD  - Department of Neurological Sciences, Rush University Medical Center, Chicago, IL, 
      USA. jkordowe@rush.edu.
AD  - The Van Andel Research Institute, Grand Rapids, MI, USA. jkordowe@rush.edu.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Review
PL  - United States
TA  - Methods Mol Biol
JT  - Methods in molecular biology (Clifton, N.J.)
JID - 9214969
RN  - 0 (HTT protein, human)
RN  - 0 (Huntingtin Protein)
RN  - 0 (Nerve Growth Factors)
SB  - IM
MH  - Animals
MH  - Cell Engineering/methods
MH  - Corpus Striatum/cytology/metabolism/*pathology
MH  - Disease Models, Animal
MH  - Drug Delivery Systems/methods
MH  - Genetic Therapy/instrumentation/*methods/trends
MH  - Genetic Vectors/*administration & dosage/genetics
MH  - Humans
MH  - Huntingtin Protein/genetics/metabolism
MH  - Huntington Disease/genetics/pathology/*therapy
MH  - Macaca fascicularis
MH  - Mice
MH  - Nerve Growth Factors/administration & dosage/*genetics
MH  - Neurons/metabolism/pathology
MH  - Rats
MH  - Stereotaxic Techniques/instrumentation
OTO - NOTNLM
OT  - Adeno-associated viral vector, AAV
OT  - Ciliary neurotrophic factor, CDNF
OT  - Encapsulated cells
OT  - Gene therapy
OT  - Glial-derived neurotrophic factor, GDNF
OT  - Neurotrophic factors
OT  - Neurturin
EDAT- 2018/06/02 06:00
MHDA- 2019/02/20 06:00
CRDT- 2018/06/02 06:00
PHST- 2018/06/02 06:00 [entrez]
PHST- 2018/06/02 06:00 [pubmed]
PHST- 2019/02/20 06:00 [medline]
AID - 10.1007/978-1-4939-7825-0_24 [doi]
PST - ppublish
SO  - Methods Mol Biol. 2018;1780:525-547. doi: 10.1007/978-1-4939-7825-0_24.