PMID- 27335539 OWN - NLM STAT- PubMed-not-MEDLINE DCOM- 20160623 LR - 20200930 IS - 1673-5374 (Print) IS - 1876-7958 (Electronic) IS - 1673-5374 (Linking) VI - 11 IP - 5 DP - 2016 May TI - Clinical trial perspective for adult and juvenile Huntington's disease using genetically-engineered mesenchymal stem cells. PG - 702-5 LID - 10.4103/1673-5374.182682 [doi] AB - Progress to date from our group and others indicate that using genetically-engineered mesenchymal stem cells (MSC) to secrete brain-derived neurotrophic factor (BDNF) supports our plan to submit an Investigational New Drug application to the Food and Drug Administration for the future planned Phase 1 safety and tolerability trial of MSC/BDNF in patients with Huntington's disease (HD). There are also potential applications of this approach beyond HD. Our biological delivery system for BDNF sets the precedent for adult stem cell therapy in the brain and could potentially be modified for other neurodegenerative disorders such as amyotrophic lateral sclerosis (ALS), spinocerebellar ataxia (SCA), Alzheimer's disease, and some forms of Parkinson's disease. The MSC/BDNF product could also be considered for studies of regeneration in traumatic brain injury, spinal cord and peripheral nerve injury. This work also provides a platform for our future gene editing studies, since we will again use MSCs to deliver the needed molecules into the central nervous system. FAU - Deng, Peter AU - Deng P AD - Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA. FAU - Torrest, Audrey AU - Torrest A AD - Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA. FAU - Pollock, Kari AU - Pollock K AD - Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA. FAU - Dahlenburg, Heather AU - Dahlenburg H AD - Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA. FAU - Annett, Geralyn AU - Annett G AD - Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA. FAU - Nolta, Jan A AU - Nolta JA AD - Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA. FAU - Fink, Kyle D AU - Fink KD AUID- ORCID: 0000-0002-0235-5038 AD - Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health System, Sacramento, CA, USA. LA - eng GR - F32 NS090722/NS/NINDS NIH HHS/United States GR - R01 GM099688/GM/NIGMS NIH HHS/United States GR - T32 GM099608/GM/NIGMS NIH HHS/United States PT - Journal Article PT - Review PL - India TA - Neural Regen Res JT - Neural regeneration research JID - 101316351 PMC - PMC4904446 OTO - NOTNLM OT - Huntington's disease OT - brain derived neurotrophic factor OT - genetic engineering OT - mesenchymal stem cells OT - neurodegenerative disorders EDAT- 2016/06/24 06:00 MHDA- 2016/06/24 06:01 PMCR- 2016/05/01 CRDT- 2016/06/24 06:00 PHST- 2016/06/24 06:00 [entrez] PHST- 2016/06/24 06:00 [pubmed] PHST- 2016/06/24 06:01 [medline] PHST- 2016/05/01 00:00 [pmc-release] AID - NRR-11-702 [pii] AID - 10.4103/1673-5374.182682 [doi] PST - ppublish SO - Neural Regen Res. 2016 May;11(5):702-5. doi: 10.4103/1673-5374.182682.