Page 20 - OR-1-2
P. 20

of lysosomal proteolysis in astrocytes facilitates midbrain   and inflammatory defects in GBA1-mutant astrocytes
                organoid proteostasis failure in an early-onset Parkinson’s   are  normalized  by  LRRK2  inhibition.  Mov  Disord.
                disease model. Nat Commun. 2024;15(1):447.       2020;35(5):760-773.
                doi: 10.1038/s41467-024-44732-2                  doi: 10.1002/mds.27994
            49.  Boussaad I, Obermaier CD, Hanss Z, et al. A patient-based   60.  Li ZF, Cui L, Jin MM, et al. A matrigel-based 3D construct
                model of RNA mis-splicing uncovers treatment targets in   of SH-SY5Y cells models the  α-synuclein pathologies of
                Parkinson’s disease. Sci Transl Med. 2020;12(560):eaau3960.  Parkinson’s disease. Dis Model Mech. 2022;15(3):dmm049125.
                doi: 10.1126/scitranslmed.aau3960                doi: 10.1242/dmm.049125
            50.  Sidransky E, Samaddar T, Tayebi N. Mutations in GBA are   61.  Chlebanowska P, Tejchman A, Sulkowski M, Skrzypek K,
                associated with familial Parkinson disease susceptibility and   Majka M. Use of 3D organoids as a model to study idiopathic
                age at onset. Neurology. 2009;73(17):1424-1425, author reply   form of Parkinson’s disease. Int J Mol Sci. 2020;21(3):694.
                1425-1426.
                                                                 doi: 10.3390/ijms21030694
                doi: 10.1212/WNL.0b013e3181b28601
                                                              62.  Elitt MS, Barbar L, Tesar PJ. Drug screening for human
            51.  Nichols WC, Pankratz N, Marek DK, et al. Mutations in GBA   genetic diseases using iPSC models.  Hum Mol Genet.
                are associated with familial Parkinson disease susceptibility   2018;27(R2):R89-R98.
                and age at onset. Neurology. 2009;72(4):310-316.
                                                                 doi: 10.1093/hmg/ddy186
                doi: 10.1212/01.wnl.0000327823.81237.d1
                                                              63.  Cetin S, Knez D, Gobec S, Kos J, Pislar A. Cell models
            52.  Rosety I, Zagare A, Saraiva C,  et al. Impaired neuron   for Alzheimer’s and Parkinson’s disease: At the interface
                differentiation in GBA-associated Parkinson’s disease is   of biology and drug discovery.  Biomed Pharmacother.
                linked to cell cycle defects in organoids. NPJ Parkinsons Dis.   2022;149:112924.
                2023;9(1):166.
                                                                 doi: 10.1016/j.biopha.2022.112924
                doi: 10.1038/s41531-023-00616-8
                                                              64.  Zhang QS, Heng Y, Mou Z, Huang JY, Yuan YH,
            53.  Zagare A, Hemedan A, Almeida C, et al. Insulin resistance   Chen NH. Reassessment of subacute MPTP-treated mice
                is a modifying factor for Parkinson’s disease. Mov Disord.   as animal model of Parkinson’s disease. Acta Pharmacol Sin.
                2024;40(1):67-76.                                2017;38(10):1317-1328.
                doi: 10.1002/mds.30039                           doi: 10.1038/aps.2017.49
            54.  Frattini E, Faustini G, Lopez G,  et al. Lewy pathology   65.  Cannon JR, Tapias V, Na HM, Honick AS, Drolet RE,
                formation in patient-derived GBA1 Parkinson’s disease   Greenamyre JT. A  highly reproducible rotenone model of
                midbrain organoids. Brain. 2025;148(4):1242-1257.  Parkinson’s disease. Neurobiol Dis. 2009;34(2):279-290.
                doi: 10.1093/brain/awae365                       doi: 10.1016/j.nbd.2009.01.016
            55.  Baden P, Perez MJ, Raji H,  et al. Glucocerebrosidase is   66.  Giasson BI, Duda JE, Quinn SM, Zhang B, Trojanowski JQ,
                imported into mitochondria and preserves complex I integrity   Lee VM. Neuronal alpha-synucleinopathy with severe
                and energy metabolism. Nat Commun. 2023;14(1):1930.  movement disorder in mice expressing A53T human alpha-
                                                                 synuclein. Neuron. 2002;34(4):521-533.
                doi: 10.1038/s41467-023-37454-4
                                                                 doi: 10.1016/s0896-6273(02)00682-7
            56.  Jo J, Yang L, Tran HD, et al. Lewy body-like inclusions in
                human midbrain organoids carrying glucocerebrosidase and   67.  Jackson-Lewis V, Przedborski S. Protocol for the MPTP mouse
                α-synuclein mutations. Ann Neurol. 2021;90(3):490-505.  model of Parkinson’s disease. Nat Protoc. 2007;2(1):141-151.
                doi: 10.1002/ana.26166                           doi: 10.1038/nprot.2006.342
            57.  Mazzulli JR, Xu YH, Sun Y,  et al. Gaucher disease   68.  Kim MS, Ra EA, Kweon SH,  et al. Advanced human
                glucocerebrosidase and  α-synuclein form a bidirectional   iPSC-based preclinical model for Parkinson’s disease with
                pathogenic loop in synucleinopathies.  Cell. 2011;   optogenetic alpha-synuclein aggregation.  Cell Stem Cell.
                146(1):37-52.                                    2023;30(7):973-986.e11.
                doi: 10.1016/j.cell.2011.06.001                  doi: 10.1016/j.stem.2023.05.015
            58.  Ysselstein  D, Nguyen  M,  Young  TJ,  et  al.  LRRK2  kinase   69.  Zhu W, Tao M, Hong Y, et al. Dysfunction of vesicular storage
                activity regulates lysosomal glucocerebrosidase in neurons   in young-onset Parkinson’s patient-derived dopaminergic
                derived  from Parkinson’s disease  patients.  Nat Commun.   neurons and organoids revealed by single cell electrochemical
                2019;10(1):5570.                                 cytometry. Chem Sci. 2022;13(21):6217-6223.
                doi: 10.1038/s41467-019-13413-w                  doi: 10.1039/d2sc00809b
            59.  Sanyal A, DeAndrade MP, Novis HS,  et al. Lysosome   70.  Jarazo J, Barmpa K, Modamio J,  et al.  Parkinson’s disease


            Volume 1 Issue 2 (2025)                         11                           doi: 10.36922/OR025040006
   15   16   17   18   19   20   21   22   23   24   25