دورية أكاديمية
أعرض في EDS

Optimizing maturity and dose of iPSC-derived dopamine progenitor cell therapy for Parkinson's disease.

التفاصيل البيبلوغرافية
العنوان: Optimizing maturity and dose of iPSC-derived dopamine progenitor cell therapy for Parkinson's disease.
المؤلفون: Hiller BM; Department of Neurological Sciences, Rush University Medical Center, Chicago, IL, USA., Marmion DJ; Department of Neurological Sciences, Rush University Medical Center, Chicago, IL, USA.; Department of Neurobiology, Barrow Neurological Institute, Phoenix, AZ, USA., Thompson CA; FUJIFILM Cellular Dynamics Inc., Madison, WI, USA., Elliott NA; FUJIFILM Cellular Dynamics Inc., Madison, WI, USA., Federoff H; Brooklyn ImmunoTherapeutics, Brooklyn, NY, USA., Brundin P; Parkinson's Disease Center, Department for Neurodegenerative Science, Van Andel Institute, Grand Rapids, MI, USA., Mattis VB; FUJIFILM Cellular Dynamics Inc., Madison, WI, USA., McMahon CW; FUJIFILM Cellular Dynamics Inc., Madison, WI, USA., Kordower JH; Department of Neurological Sciences, Rush University Medical Center, Chicago, IL, USA. jeffrey.kordower@asu.edu.; ASU-Banner Neurodegenerative Disease Research Center and School of Life Sciences, Arizona State University, Tempe, AZ, USA. jeffrey.kordower@asu.edu.
المصدر: NPJ Regenerative medicine [NPJ Regen Med] 2022 Apr 21; Vol. 7 (1), pp. 24. Date of Electronic Publication: 2022 Apr 21.
نوع المنشور: Journal Article
اللغة: English
بيانات الدورية: Publisher: Nature Publishing Group, published in partnership with Monash University and Australian Regenerative Medicine Institute Country of Publication: United States NLM ID: 101699846 Publication Model: Electronic Cited Medium: Internet ISSN: 2057-3995 (Electronic) Linking ISSN: 20573995 NLM ISO Abbreviation: NPJ Regen Med Subsets: PubMed not MEDLINE
أسماء مطبوعة: Original Publication: [New York] : Nature Publishing Group, published in partnership with Monash University and Australian Regenerative Medicine Institute, [2016]-
مستخلص: In pursuit of treating Parkinson's disease with cell replacement therapy, differentiated induced pluripotent stem cells (iPSC) are an ideal source of midbrain dopaminergic (mDA) cells. We previously established a protocol for differentiating iPSC-derived post-mitotic mDA neurons capable of reversing 6-hydroxydopamine-induced hemiparkinsonism in rats. In the present study, we transitioned the iPSC starting material and defined an adapted differentiation protocol for further translation into a clinical cell transplantation therapy. We examined the effects of cellular maturity on survival and efficacy of the transplants by engrafting mDA progenitors (cryopreserved at 17 days of differentiation, D17), immature neurons (D24), and post-mitotic neurons (D37) into immunocompromised hemiparkinsonian rats. We found that D17 progenitors were markedly superior to immature D24 or mature D37 neurons in terms of survival, fiber outgrowth and effects on motor deficits. Intranigral engraftment to the ventral midbrain demonstrated that D17 cells had a greater capacity than D24 cells to innervate over long distance to forebrain structures, including the striatum. When D17 cells were assessed across a wide dose range (7,500-450,000 injected cells per striatum), there was a clear dose response with regards to numbers of surviving neurons, innervation, and functional recovery. Importantly, although these grafts were derived from iPSCs, we did not observe teratoma formation or significant outgrowth of other cells in any animal. These data support the concept that human iPSC-derived D17 mDA progenitors are suitable for clinical development with the aim of transplantation trials in patients with Parkinson's disease.
(© 2022. The Author(s).)
References: Cell. 2006 Aug 25;126(4):663-76. (PMID: 16904174)
Exp Cell Res. 1999 Dec 15;253(2):737-46. (PMID: 10585298)
Front Cell Neurosci. 2014 Sep 09;8:275. (PMID: 25249938)
JAMA Neurol. 2018 Jan 1;75(1):9-10. (PMID: 29131880)
Regen Med. 2010 Sep;5(5):787-97. (PMID: 20868333)
J Clin Invest. 2012 Aug;122(8):2928-39. (PMID: 22751106)
Ann Neurol. 2003 Sep;54(3):403-14. (PMID: 12953276)
J Neurosci Methods. 2009 Jan 30;176(2):200-5. (PMID: 18809434)
Cell Stem Cell. 2021 Feb 4;28(2):343-355.e5. (PMID: 33545081)
Exp Brain Res. 1986;65(1):235-40. (PMID: 3542544)
Cell Transplant. 1995 Jan-Feb;4(1):141-54. (PMID: 7728329)
Cell Stem Cell. 2015 Mar 5;16(3):269-74. (PMID: 25732245)
Brain. 2009 Feb;132(Pt 2):319-35. (PMID: 19039008)
J Comp Neurol. 2018 Sep 1;526(13):2133-2146. (PMID: 30007046)
Cell Rep. 2012 Jun 28;1(6):703-14. (PMID: 22813745)
Mov Disord. 2021 Aug;36(8):1772-1780. (PMID: 33963552)
Mov Disord. 2009 Feb 15;24(3):336-43. (PMID: 19006186)
Front Cell Dev Biol. 2021 May 13;9:666675. (PMID: 34055800)
Ann Neurol. 1995 Sep;38(3):379-88. (PMID: 7668823)
Science. 1998 Nov 6;282(5391):1145-7. (PMID: 9804556)
Methods Mol Biol. 2013;1018:11-9. (PMID: 23681613)
Development. 2017 Mar 1;144(5):916-927. (PMID: 28174244)
Nat Rev Neurol. 2015 Sep;11(9):492-503. (PMID: 26240036)
Exp Neurol. 1989 Oct;106(1):1-19. (PMID: 2477271)
Stem Cell Reports. 2017 Jul 11;9(1):149-161. (PMID: 28579395)
Nat Commun. 2020 Jul 6;11(1):3369. (PMID: 32632153)
Exp Neurol. 2008 Sep;213(1):220-8. (PMID: 18602916)
Stem Cells Transl Med. 2021 Feb;10(2):278-290. (PMID: 32997443)
PLoS Biol. 2007 Dec;5(12):e325. (PMID: 18076286)
Trends Neurosci. 2020 Mar;43(3):155-169. (PMID: 32101709)
Brain Res Bull. 2005 Dec 15;68(1-2):4-15. (PMID: 16324999)
Cell Stem Cell. 2014 Nov 6;15(5):653-65. (PMID: 25517469)
Nat Med. 2019 Jul;25(7):1045-1053. (PMID: 31263283)
N Engl J Med. 2001 Mar 8;344(10):710-9. (PMID: 11236774)
Ann Neurol. 2002 Nov;52(5):628-34. (PMID: 12402261)
Nature. 2011 Nov 06;480(7378):547-51. (PMID: 22056989)
Cell Stem Cell. 2021 Feb 4;28(2):217-229.e7. (PMID: 33545080)
Stem Cells Transl Med. 2017 Mar;6(3):937-948. (PMID: 28297587)
Stem Cell Reports. 2015 Jun 9;4(6):975-83. (PMID: 26004633)
Exp Neurol. 2012 Jul;236(1):58-68. (PMID: 22524988)
Science. 2001 Mar 16;291(5511):2147-50. (PMID: 11251119)
Cell Stem Cell. 2017 Jan 5;20(1):29-40. (PMID: 28094018)
Mov Disord. 2001 May;16(3):448-58. (PMID: 11391738)
Stem Cells Transl Med. 2017 Sep;6(9):1803-1814. (PMID: 28650520)
Cell Stem Cell. 2017 Jan 5;20(1):135-148. (PMID: 28094017)
J Clin Invest. 2020 Feb 3;130(2):904-920. (PMID: 31714896)
J Neurosci. 2005 Jul 6;25(27):6467-77. (PMID: 16000637)
J Comp Neurol. 2012 Aug 15;520(12):2591-607. (PMID: 22252428)
Primate Biol. 2017 Oct 11;4(2):185-213. (PMID: 32110706)
Science. 1990 Feb 2;247(4942):574-7. (PMID: 2105529)
Nat Neurosci. 2002 Jul;5(7):627-8. (PMID: 12042822)
Neuron. 2015 Apr 8;86(1):187-206. (PMID: 25856494)
تواريخ الأحداث: Date Created: 20220422 Latest Revision: 20220716
رمز التحديث: 20240829
مُعرف محوري في PubMed: PMC9023503
DOI: 10.1038/s41536-022-00221-y
PMID: 35449132
قاعدة البيانات: MEDLINE
الوصف
تدمد:2057-3995
DOI:10.1038/s41536-022-00221-y