US 5789538A
· Rebar et al.
· 1998
[cited by applicant]
US 5925923A
· Blair
· 1999
[cited by applicant]
US 6007988A
· Choo et al.
· 1999
[cited by applicant]
US 6013453A
· Choo et al.
· 2000
[cited by applicant]
US 6140466A
· Barbas et al.
· 2000
[cited by applicant]
US 6200759B1
· Dove et al.
· 2001
[cited by applicant]
US 6242568B1
· Barbas et al.
· 2001
[cited by applicant]
US 6410248B1
· Greisman et al.
· 2002
[cited by applicant]
US 6453242B1
· Eisenberg et al.
· 2002
[cited by applicant]
US 6479626B1
· Kim et al.
· 2002
[cited by applicant]
US 6534261B1
· Cox et al.
· 2003
[cited by applicant]
US 6607882B1
· Cox et al.
· 2003
[cited by applicant]
US 6903185B2
· Kim et al.
· 2005
[cited by applicant]
US 7153949B2
· Kim et al.
· 2006
[cited by applicant]
US 8492145B2
· Nakayama et al.
· 2013
[cited by applicant]
US 20080274446A1
· Stice et al.
· 2008
[cited by applicant]
US 20160298080A1
· Ying et al.
· 2016
[cited by applicant]
US 20200018746A1
· Tekin et al.
· 2020
[cited by applicant]
US 20200056149A1
· Tomishima et al.
· 2020
[cited by applicant]
US 20200191776A1
· Eggan
· 2020
[cited by examiner]
WO 2018165577A1
· 2018
[cited by applicant]
WO WO2021086063A1
· 2021
[cited by examiner]
Schmid, B., et al., “Generation of two gene edited iPSC-lines carrying a DOX-inducible NGN2 expression cassette with and without GFP in the AAVS1 locus,” Stem Cell Research 52: 102240. doi: 10.1016/j.scr.2021.102240. Ep…
[cited by examiner]
Shih, P. Y., et al., “Development of a fully human assay combining NGN2-inducible neurons co-cultured with iPSC-derived astrocytes amenable for electrophysiological studies,” Stem Cell Research 54: 102386. doi: 10.1016/…
[cited by examiner]
Deneault, E., et al., “Complete Disruption of Autism-Susceptibility Genes by Gene Editing Predominantly Reduces Functional Connectivity of Isogenic Human Neurons,” Stem Cell Reports 11(5): 1211-1225. doi: 10.1016/j.stem…
[cited by examiner]
Sagal et al., “Proneural transcription factor Atoh1 drives highly efficient differentiation of human pluripotent stem cells into dopaminergic neurons,” Stem Cells Translational Medicine 3: 888-898 (Year: 2014).
[cited by examiner]
Baker, N. E., and Brown, “All in the family: proneural bHLH genes and neuronal diversity,” Development 145(9): dev159426. doi: 10.1242/dev.159426. (Year: 2018).
[cited by examiner]
Serra, M., et al., “Microencapsulation technology: a powerful tool for integrating expansion and cryopreservation of human embryonic stem cells,” PLoS One 6(8): e23212. doi: 10.1371/journal.pone.0023212. (Year: 2011).
[cited by examiner]
Hall, P.E., et al., “Laminin enhances the growth of human neural stem cells in defined culture media,” BMC Neurosci 9: 71. doi: 10.1186/1471-2202-9-71. (Year: 2008).
[cited by examiner]
Bardy, C. et al., “Neuronal medium that supports basic synaptic functions and activity of human neurons in vitro,” Proc Natl Acad Sci USA 112(20): E2725-E2734. doi: 10.1073/pnas.1504393112. (Year: 2015).
[cited by examiner]
Kampmann, M., “CRISPR-based functional genomics for neurological disease,” Nat Rev Neurol 16(9):465-480. doi: 10.1038/s41582-020-0373-z. Epub Jul. 8, 2020. (Year: 2020).
[cited by examiner]
Engle, S. J., et al., “Best Practices for Translational Disease Modeling Using Human iPSC-Derived Neurons,” Neuron 100(4):783-797. doi: 10.1016/j.neuron.2018.10.033. (Year: 2018).
[cited by examiner]
Alami , et al., “Microtubule-dependent transport of TDP-43 mRNA granules in neurons is impaired by ALS⋅causing mutations”, Neuron 81(3):536-543.
[cited by applicant]
Almeida , et al., “Modeling key pathological features of frontotemporal dementia with C9ORF72 repeat expansion in iPSC-derived human neurons”, Acta Neuropathol 126(3):385-399.
[cited by applicant]
Amorso , et al., “Accelerated high-yield generation of limb-innervating motor neurons from human stem cells”, J Neurosci 33(2):574-86.
[cited by applicant]
An , et al., “Genetic correction of Huntington's disease phenotypes in induced pluripotent stem cells”, Cell Stem Cell 11(2):253-263.
[cited by applicant]
Ananiev , et al., “Isogenic pairs of wild type and mutant induced pluripotent stem cell (iPSC) lines from Rett syndrome patients as in vitro disease model”, PLoS One 6(9):e25255.
[cited by applicant]
Andrade , et al., “Evidence for premature aging due to oxidative stress in iPSCs from Cockayne syndrome”, Hum Mol Genet 21:3825-3834 (2012).
[cited by applicant]
Bardy , et al., “Neuronal medium that supports basic synaptic functions and activity of human neurons in vitro”, Proc Natl Acad Sci USA 112:E2725-34 (2015).
[cited by applicant]
Bilican , et al., “Mutant induced pluripotent stem cell lines recapitulate aspects of TDP-43 proteinopathies and reveal cell-specific vulnerability”, PNAS 109(15):5803-5808.
[cited by applicant]
Boulting , et al., “A functionally characterized test set of human induced pluripotent stem cells”, Nat Biotech 29(3):279-286.
[cited by applicant]
Brennand , et al., “Modelling schizophrenia using human induced pluripotent stem cells,”, Nature 473 (7346):221-225.
[cited by applicant]
Camnasio , et al., “The first reported generation of several induced pluripotent stem cell lines from homozygous and heterozygous Huntington's disease patients demonstrates mutation related enhanced lysosomal activity”,…
[cited by applicant]
Chiang , et al., “Integration⋅free induced pluripotent stem cells derived from schizophrenia patients with a Disc 1 mutation”, Molecular Psych 16:358-360.
[cited by applicant]
Consortium , et al., “Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes”, Cell Stem Cell 11(2):264-278.
[cited by applicant]
Corti , et al., “Genetic correction of human induced pluripotent stem cells from patients with spinal muscular atrophy,”, Sci Transl Med 4 (165): I 65ral 62.
[cited by applicant]
Deneault , et al., “Complete Disruption of Autism-Susceptibility Genes by Gene Editing Predominantly Reduces Functional Connectivity of Isogenic Human Neurons”, Stem Cell Reports 11:1211-25 (2018).
[cited by applicant]
Denton , et al., “Loss of spastin function results in disease-specific axonal defects in human pluripotent stem cel ⋅⋅ based models of hereditary spastic paraplegia”, Stem Cells 32(2):414-23.
[cited by applicant]
Dimos , et al., “Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons”, Science 321(5893): 1218-2.
[cited by applicant]
Donnelly , et al., “RNA toxicity from the ALS/FTD C9orf72 expansion is mitigated by antisense intervention”, Neuron 80(2):415-28.
[cited by applicant]
Du , et al., “Role of Mismatch repair enzymes in GAA-TTC triplet-repeat expansion in Friedreich ataxia induced pluripotent stem cells”, J Biol Chem 287(35):29861-29872 (2012).
[cited by applicant]
Ebert , et al., “Induced pluripotent stem cells from a spinal muscular atrophy patient”, Nature 457(7227):277-80.
[cited by applicant]
Egawa , et al., “Drug screening for ALS using patient-specific induced pluripotent stem cells”, Sci Transl Med 4(145):145ral04.
[cited by applicant]
Fong , et al., “Genetic correction of tauopathy phenotypes in neurons derived from human induced pluripotent stem cells”, Stem Cell Reports 1(3): 1-9.
[cited by applicant]
Hall , et al., “Laminin enhances the growth of human neural stem cells in defined culture media”, BMC Neurosci 9:71 (2008).
[cited by applicant]
Han , et al., “Constructing and deconstructing stem cell models of neurological disease”, Neuron 70(4):626-44.
[cited by applicant]
Harel and Lupski , “Charcot Marie Tooth disease and pathways to molecular based therapies,”, Clin Genet DOI: 10.1111/cge.12393.
[cited by applicant]
Hick , et al., “Neurons and cardiomyocytes derived from induced pluripotent stem cells as a model from mitochondrial defects in Friedreich's ataxia”, Dis Model Mech 6(3):608-21 (2013).
[cited by applicant]
Higurashi , “A human Dravet syndrome model from patient induced pluripotent stem cells”, Mol Brain 6:19.
[cited by applicant]
Israel , et al., “Probing sporadic and familial Alzheimer's disease using induced pluripotent stem cells,”, Nature 482 (7384):216-20.
[cited by applicant]
Kiskinis , et al., “Pathways disrupted in human ALS motor neurons identified through genetic correction of mutant SODI”, Cell Stem Cell (epub).
[cited by applicant]
Koch , et al., “Excitation-induced ataxin-3 aggregation in neurons from patients with Machado-Joseph disease”, Nature 480(7378):543-546.
[cited by applicant]
Kondo , et al., “Modeling Alzheimer's disease with iPSCs reveals stress phenotypes associated with intracellular Abeta and differential drug responsiveness”, Cell Stem Cell 12(4):487-496.
[cited by applicant]
Kray , et al., “Timothy syndrome is associated with activity-dependent dendritic retraction in rodent and human neurons,”, Nat Neurosci 16(2):201-9.
[cited by applicant]
Ku , et al., “Friedreich's ataxia induced pluripotent stem cells model intergenerational GAA-TTC triplet repeat instability”, Cell Stem Cell 7(5):631-7.
[cited by applicant]
Lee , et al., “Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs”, Nature, 461:402-406 (2009).
[cited by applicant]
Liu , et al., “Signaling defects in iPSC-derived fragile X premutation neurons”, Hum Mol Genet 21:3795-3805 (2012).
[cited by applicant]
Mahammad , et al., “Giant axonal neuropathy-associated gigaxonin mutations impair intermediate filament protein degredation,”, J Clin Invest 123(5): 1964-75.
[cited by applicant]
Marchetto , et al., “A model for neural development and treatment of Rett syndrome using human induced pluripotent stem cells”, Cell, 143(4):527-39.
[cited by applicant]
Mazzulli , et al., “Gaucher disease glucocerebrosidase and a-synuclein form a bidirectional pathogenic loop in synucleinopathies”, Cell 146(1):37-52 (2011).
[cited by applicant]
Muratore , et al., “The familial Alzheimer's disease APPV7171 mutation alters APP processing and tau expression in iPSC-derived neurons”, Human Molecular Genetics, in press.
[cited by applicant]
Nihei , et al., “Enhanced aggregation of androgen receptor in induced pluripotent stem cell-derived neurons from spinal and bulbar muscular atrophy,”, J Biol Chem 288(12):8043-52.
[cited by applicant]
Sareen , et al., “Inhibition of apoptosis blocks human motor neuron cell death in a stem cell model of spinal muscular atrophy”, PLoS One 7(6):e39113.
[cited by applicant]
Serra , et al., “Microencapsulation Technology: A Powerful Tool for Integrating Expansion and Cryopreservation of Human Embryonic Stem Cells”, PLoS One 6:e23212 (2011).
[cited by applicant]
Shcheglovitov , et al., “SHANK3 and IGFI restore synaptic deficits in neurons from 22q 13 deletion syndrome patients”, Nature 503(7475):267-71.
[cited by applicant]
Shi , et al., “A human stem cell model of early Alzheimer's disease pathology in Down syndrome”, Sci Transl Med 4(124):124ra129.
[cited by applicant]
Shih , et al., “Development of a fully human assay combining NGN2-inducible neurons co-cultured with iPSC-derived astrocytes amenable for electrophysiological studies”, Stem Cell Res 54:102386 (2021).
[cited by applicant]
Song, et al., “Neural differentiation of patient specific iPS cells as a novel approach to study the pathophysiology of multiple sclerosis”, Stem Cell Res 8(2):259-73.
[cited by applicant]
Wainger , et al., “Intrinsic membrane hyperexcitability of amyotrophic lateral sclerosis patient-derived motor neurons”, Cell Reports 7(1): 1-11.
[cited by applicant]
Yang , et al., “A small molecule screen in stem-cell-derived motor neurons identifies a kinase inhibitor as a candidate therapeutic for ALS”, Cell Stem Cell 12(6):713-726.
[cited by applicant]
Zhang , et al., “Rapid single-step induction of functional neurons from human pluripotent stem cells”, Neuron 78(5):785-798.
[cited by applicant]
Liu et al, 2012, Efficient and specific modifications of the
[cited by applicant]
Joung & Sander, 2013, TALENs: a widely applicable technology for targeted genome editing, Nat Rev Mol Cell Bio 14:49-55.
[cited by applicant]
Chang et al., 2013, Genome editing with RNA⋅guided Cas9 nuclease in zebrafish embryos, Cell Res 23 :465-472.
[cited by applicant]
Hwang et al., 2013, Efficient genome editing in zebrafish using a CRISPR-Cas system, Nat. Biotechnol 31 :227-229.
[cited by applicant]
Xiao et al., 2013, Chromosomal deletions and inversions mediated by TALENS and CRISPR/Cas in zebrafish, Nucl Acids Res 1-11.
[cited by applicant]
Beerli & Barbas, 2002, Engineering polydactyl zinc-finger transcription factors, Nat. Biotechnol, 20: 135-141.
[cited by applicant]
Pabo et al., 2001, Design and selection of novel Cys2His2 zinc finger proteins, Ann. Rev. Biochem 70:313-340.
[cited by applicant]
Isalan et al., 2001, A rapid generally applicable method to engineer zinc fingers illustrated by targeting the HIV-I promoter, Nat. Biotechnol 19:656-660.
[cited by applicant]
Santiago et al., 2008, Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases, PNAS 105:5809-5814.
[cited by applicant]
Sakai et al., 2001, Design and characterization of a DNA encoded, voltage-sensitive fluorescent protein, Euro J Neuroscience 13 :2314-2318.
[cited by applicant]
Murata et al., 2005, Phosphoinositide phosphatase activity coupled to an intrinsic voltage sensor, Nature 435:1239-1243.
[cited by applicant]
Chanda et al., 2005, A hybrid approach to measuring electrical activity in genetically specified neurons, Nat Neuroscience 8: 1619-1626.
[cited by applicant]
Kralj et al, 2012, Optical recording of action potentials in mammalian neurons using a microbial rhodopsin, Nat Methods 9:90-95.
[cited by applicant]
Carlson and Campbell, 2013, Circular permutated red fluorescent proteins and calcium ion indicators based on mCherry, Protein Eng Des Sel 26(12):763-772.
[cited by applicant]
Klapoetke et al., 2014, Independent optical excitation of distinct neural populations, Nat Meth Advance Online Publication 1-14.
[cited by applicant]
Gingrich and Roder, 1998, Inducible gene expression in the nervous system of transgenic mice, Annu Rev Neurosci 21 :377-405.
[cited by applicant]
Wardill et al., 2013, A neuron-based screening platform for optimizing genetically-encoded calcium indicators, PLoS One 8(10):e77728.
[cited by applicant]
Dottori, et al., Neural development in human embryonic stem cells-applications of lentiviral vectors, J Cell Biochem 112(8): 1955-62.
[cited by applicant]
Diester et al., 2011, An optogenetic toolbox designed for primates, Nat Neurosci 14(3):387-97.
[cited by applicant]
Atasoy et al., 2009, A Flex switch targets channelrhodopsin-2 to multiple cell types for imaging and Ion⋅⋅range circuit mapping, J Neurosci 28(28):7025-7030.
[cited by applicant]
Rothermel et al., 2013, Transgene expression in target-defined neuron populations mediated by retrograde infection with adeno-associated viral vectors, J Neurosci 33(38): 195-206.
[cited by applicant]
Williams et al.: “Scalable Measurements of Intrinsic Excitability in Human iPS Cell-Derived Excitatory Neurons Using All-Optical Electrophysiology”, Neurochemical Research, (2019), vol. 44, pp. 714-725.
[cited by applicant]
Zhang et al.: “Chronic optogenetic induction of stress granules is cytotoxic and reveals the evolution of ALS-FTD pathology”, eLife, (2019), vol. 8, e39578, pp. 1-23.
[cited by applicant]