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CGG-repeat dynamics and FMR1 gene silencing in fragile X syndrome stem cells and stem cell-derived neurons / Y. ZHOU in Molecular Autism, 7 (2016)
[article]
Titre : CGG-repeat dynamics and FMR1 gene silencing in fragile X syndrome stem cells and stem cell-derived neurons Type de document : Texte imprimé et/ou numérique Auteurs : Y. ZHOU, Auteur ; D. KUMARI, Auteur ; N. SCIASCIA, Auteur ; K. USDIN, Auteur Article en page(s) : 42p. Langues : Anglais (eng) Mots-clés : 5' Untranslated Regions Alleles Cell Differentiation Cell Line DNA Methylation Embryonic Stem Cells/metabolism/pathology Fragile X Mental Retardation Protein/genetics/metabolism Fragile X Syndrome/genetics/metabolism/pathology Gene Silencing Humans Induced Pluripotent Stem Cells/metabolism/pathology Male Neurons/metabolism/pathology Primary Cell Culture Time Factors Trinucleotide Repeat Expansion Fragile X syndrome Repeat contractions Repeat expansion mutation Repeat-mediated gene silencing Stem cells Index. décimale : PER Périodiques Résumé : BACKGROUND: Fragile X syndrome (FXS), a common cause of intellectual disability and autism, results from the expansion of a CGG-repeat tract in the 5' untranslated region of the FMR1 gene to >200 repeats. Such expanded alleles, known as full mutation (FM) alleles, are epigenetically silenced in differentiated cells thus resulting in the loss of FMRP, a protein important for learning and memory. The timing of repeat expansion and FMR1 gene silencing is controversial. METHODS: We monitored the repeat size and methylation status of FMR1 alleles with expanded CGG repeats in patient-derived induced pluripotent stem cells (iPSCs) and embryonic stem cells (ESCs) that were grown for extended period of time either as stem cells or differentiated into neurons. We used a PCR assay optimized for the amplification of large CGG repeats for sizing, and a quantitative methylation-specific PCR for the analysis of FMR1 promoter methylation. The FMR1 mRNA levels were analyzed by qRT-PCR. FMRP levels were determined by western blotting and immunofluorescence. Chromatin immunoprecipitation was used to study the association of repressive histone marks with the FMR1 gene in FXS ESCs. RESULTS: We show here that while FMR1 gene silencing can be seen in FXS embryonic stem cells (ESCs), some silenced alleles contract and when the repeat number drops below ~400, DNA methylation erodes, even when the repeat number remains >200. The resultant active alleles do not show the large step-wise expansions seen in stem cells from other repeat expansion diseases. Furthermore, there may be selection against large active alleles and these alleles do not expand further or become silenced on neuronal differentiation. CONCLUSIONS: Our data support the hypotheses that (i) large expansions occur prezygotically or in the very early embryo, (ii) large unmethylated alleles may be deleterious in stem cells, (iii) methylation can occur on alleles with >400 repeats very early in embryogenesis, and (iv) expansion and contraction may occur by different mechanisms. Our data also suggest that the threshold for stable methylation of FM alleles may be higher than previously thought. A higher threshold might explain why some carriers of FM alleles escape methylation. It may also provide a simple explanation for why silencing has not been observed in mouse models with >200 repeats. En ligne : http://dx.doi.org/10.1186/s13229-016-0105-9 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=329
in Molecular Autism > 7 (2016) . - 42p.[article] CGG-repeat dynamics and FMR1 gene silencing in fragile X syndrome stem cells and stem cell-derived neurons [Texte imprimé et/ou numérique] / Y. ZHOU, Auteur ; D. KUMARI, Auteur ; N. SCIASCIA, Auteur ; K. USDIN, Auteur . - 42p.
Langues : Anglais (eng)
in Molecular Autism > 7 (2016) . - 42p.
Mots-clés : 5' Untranslated Regions Alleles Cell Differentiation Cell Line DNA Methylation Embryonic Stem Cells/metabolism/pathology Fragile X Mental Retardation Protein/genetics/metabolism Fragile X Syndrome/genetics/metabolism/pathology Gene Silencing Humans Induced Pluripotent Stem Cells/metabolism/pathology Male Neurons/metabolism/pathology Primary Cell Culture Time Factors Trinucleotide Repeat Expansion Fragile X syndrome Repeat contractions Repeat expansion mutation Repeat-mediated gene silencing Stem cells Index. décimale : PER Périodiques Résumé : BACKGROUND: Fragile X syndrome (FXS), a common cause of intellectual disability and autism, results from the expansion of a CGG-repeat tract in the 5' untranslated region of the FMR1 gene to >200 repeats. Such expanded alleles, known as full mutation (FM) alleles, are epigenetically silenced in differentiated cells thus resulting in the loss of FMRP, a protein important for learning and memory. The timing of repeat expansion and FMR1 gene silencing is controversial. METHODS: We monitored the repeat size and methylation status of FMR1 alleles with expanded CGG repeats in patient-derived induced pluripotent stem cells (iPSCs) and embryonic stem cells (ESCs) that were grown for extended period of time either as stem cells or differentiated into neurons. We used a PCR assay optimized for the amplification of large CGG repeats for sizing, and a quantitative methylation-specific PCR for the analysis of FMR1 promoter methylation. The FMR1 mRNA levels were analyzed by qRT-PCR. FMRP levels were determined by western blotting and immunofluorescence. Chromatin immunoprecipitation was used to study the association of repressive histone marks with the FMR1 gene in FXS ESCs. RESULTS: We show here that while FMR1 gene silencing can be seen in FXS embryonic stem cells (ESCs), some silenced alleles contract and when the repeat number drops below ~400, DNA methylation erodes, even when the repeat number remains >200. The resultant active alleles do not show the large step-wise expansions seen in stem cells from other repeat expansion diseases. Furthermore, there may be selection against large active alleles and these alleles do not expand further or become silenced on neuronal differentiation. CONCLUSIONS: Our data support the hypotheses that (i) large expansions occur prezygotically or in the very early embryo, (ii) large unmethylated alleles may be deleterious in stem cells, (iii) methylation can occur on alleles with >400 repeats very early in embryogenesis, and (iv) expansion and contraction may occur by different mechanisms. Our data also suggest that the threshold for stable methylation of FM alleles may be higher than previously thought. A higher threshold might explain why some carriers of FM alleles escape methylation. It may also provide a simple explanation for why silencing has not been observed in mouse models with >200 repeats. En ligne : http://dx.doi.org/10.1186/s13229-016-0105-9 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=329 Do the Stem Cells Really Work with Autism Spectrum Disorders Associated with Neuro-Immune Interaction? / Gao SHANE in Autism - Open Access, 5-3 ([01/06/2015])
[article]
Titre : Do the Stem Cells Really Work with Autism Spectrum Disorders Associated with Neuro-Immune Interaction? Type de document : Texte imprimé et/ou numérique Auteurs : Gao SHANE, Auteur ; Xu JUN, Auteur ; Wang JUAN, Auteur ; Wu ZEYANG, Auteur ; Yuan PING, Auteur ; Gao FENGJUAN, Auteur ; Cao LIMEI, Auteur ; Chen XU, Auteur ; Zhou FEI, Auteur ; Zhu HONGWEN, Auteur Article en page(s) : 7 p. Langues : Anglais (eng) Mots-clés : Stem cells Mesenchymal stem cells (Mscs) Autism spectrum disorders (Asds) Neuro-immune interaction Index. décimale : PER Périodiques Résumé : Autism spectrum disorders (ASDs), namely neurodevelopmental disorders encompassing impairments in communication, social interactions and restricted stereotypical behaviors, induces a relatively high morbidity and mortality ratio (1/166) in modern children's life. One of the serious factors accounting for ASDs is the failure of the appropriate neuro-immune interaction. Although a relationship between altered immune responses and ASDs was firstly recognized nearly 40 years ago, only recently has new evidence started to shed light on the complex multifaceted communication between neuro-immune dysfunction and behavior in ASDs. Extensive alterations in immune function have now been described in both children and adults with ASDs, including ongoing inflammation in brain specimens, elevated pro-inflammatory cytokine profiles in the Cerebro-Spinal Fluid (CSF) and blood, increased presence of brain-specific auto-antibodies and altered immune cell function. Accumulated data both from clinical and lab research proposed the essential role of neuro-immune interaction during the pathogenesis of ASDs. Stem cells, which account for normal turnover and injury repair, might do great favors on ASDs due to their ability to give rise to new functional cells as a cell replacement source, paracrine secretion as trophic and cytokine contributor, immune modulator to balance the pro-inflammation and anti-inflammation as well as the inhibitor of chronic inflammation in ASDs brain, etc. Here in this review, we focus on the current development of stem cell administration in ASDs especially on mesenchymal stem cells (MSCs), which proved to be the most plastic and efficient to interfere with ASDs neuro-immune interaction, moreover summarize the propbable mechanism and efficient therapeutic methods to treat ASDs with MSCs. En ligne : https://dx.doi.org/10.4172/2165-7890.1000151 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=409
in Autism - Open Access > 5-3 [01/06/2015] . - 7 p.[article] Do the Stem Cells Really Work with Autism Spectrum Disorders Associated with Neuro-Immune Interaction? [Texte imprimé et/ou numérique] / Gao SHANE, Auteur ; Xu JUN, Auteur ; Wang JUAN, Auteur ; Wu ZEYANG, Auteur ; Yuan PING, Auteur ; Gao FENGJUAN, Auteur ; Cao LIMEI, Auteur ; Chen XU, Auteur ; Zhou FEI, Auteur ; Zhu HONGWEN, Auteur . - 7 p.
Langues : Anglais (eng)
in Autism - Open Access > 5-3 [01/06/2015] . - 7 p.
Mots-clés : Stem cells Mesenchymal stem cells (Mscs) Autism spectrum disorders (Asds) Neuro-immune interaction Index. décimale : PER Périodiques Résumé : Autism spectrum disorders (ASDs), namely neurodevelopmental disorders encompassing impairments in communication, social interactions and restricted stereotypical behaviors, induces a relatively high morbidity and mortality ratio (1/166) in modern children's life. One of the serious factors accounting for ASDs is the failure of the appropriate neuro-immune interaction. Although a relationship between altered immune responses and ASDs was firstly recognized nearly 40 years ago, only recently has new evidence started to shed light on the complex multifaceted communication between neuro-immune dysfunction and behavior in ASDs. Extensive alterations in immune function have now been described in both children and adults with ASDs, including ongoing inflammation in brain specimens, elevated pro-inflammatory cytokine profiles in the Cerebro-Spinal Fluid (CSF) and blood, increased presence of brain-specific auto-antibodies and altered immune cell function. Accumulated data both from clinical and lab research proposed the essential role of neuro-immune interaction during the pathogenesis of ASDs. Stem cells, which account for normal turnover and injury repair, might do great favors on ASDs due to their ability to give rise to new functional cells as a cell replacement source, paracrine secretion as trophic and cytokine contributor, immune modulator to balance the pro-inflammation and anti-inflammation as well as the inhibitor of chronic inflammation in ASDs brain, etc. Here in this review, we focus on the current development of stem cell administration in ASDs especially on mesenchymal stem cells (MSCs), which proved to be the most plastic and efficient to interfere with ASDs neuro-immune interaction, moreover summarize the propbable mechanism and efficient therapeutic methods to treat ASDs with MSCs. En ligne : https://dx.doi.org/10.4172/2165-7890.1000151 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=409 Significant transcriptional changes in 15q duplication but not Angelman syndrome deletion stem cell-derived neurons / N. URRACA in Molecular Autism, 9 (2018)
[article]
Titre : Significant transcriptional changes in 15q duplication but not Angelman syndrome deletion stem cell-derived neurons Type de document : Texte imprimé et/ou numérique Auteurs : N. URRACA, Auteur ; K. HOPE, Auteur ; A. K. VICTOR, Auteur ; T. G. BELGARD, Auteur ; R. MEMON, Auteur ; S. GOORHA, Auteur ; C. VALDEZ, Auteur ; Q. T. TRAN, Auteur ; S. SANCHEZ, Auteur ; J. RAMIREZ, Auteur ; M. DONALDSON, Auteur ; D. BRIDGES, Auteur ; L. T. REITER, Auteur Article en page(s) : 6p. Langues : Anglais (eng) Mots-clés : Autism Genomic disorders Neurogenetic syndrome Stem cells mRNAseq Index. décimale : PER Périodiques Résumé : Background: The inability to analyze gene expression in living neurons from Angelman (AS) and Duplication 15q (Dup15q) syndrome subjects has limited our understanding of these disorders at the molecular level. Method: Here, we use dental pulp stem cells (DPSC) from AS deletion, 15q Duplication, and neurotypical control subjects for whole transcriptome analysis. We identified 20 genes unique to AS neurons, 120 genes unique to 15q duplication, and 3 shared transcripts that were differentially expressed in DPSC neurons vs controls. Results: Copy number correlated with gene expression for most genes across the 15q11.2-q13.1 critical region. Two thirds of the genes differentially expressed in 15q duplication neurons were downregulated compared to controls including several transcription factors, while in AS differential expression was restricted primarily to the 15q region. Here, we show significant downregulation of the transcription factors FOXO1 and HAND2 in neurons from 15q duplication, but not AS deletion subjects suggesting that disruptions in transcriptional regulation may be a driving factor in the autism phenotype in Dup15q syndrome. Downstream analysis revealed downregulation of the ASD associated genes EHPB2 and RORA, both genes with FOXO1 binding sites. Genes upregulated in either Dup15q cortex or idiopathic ASD cortex both overlapped significantly with the most upregulated genes in Dup15q DPSC-derived neurons. Conclusions: Finding a significant increase in both HERC2 and UBE3A in Dup15q neurons and significant decrease in these two genes in AS deletion neurons may explain differences between AS deletion class and UBE3A specific classes of AS mutation where HERC2 is expressed at normal levels. Also, we identified an enrichment for FOXO1-regulated transcripts in Dup15q neurons including ASD-associated genes EHPB2 and RORA indicating a possible connection between this syndromic form of ASD and idiopathic cases. En ligne : http://dx.doi.org/10.1186/s13229-018-0191-y Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=354
in Molecular Autism > 9 (2018) . - 6p.[article] Significant transcriptional changes in 15q duplication but not Angelman syndrome deletion stem cell-derived neurons [Texte imprimé et/ou numérique] / N. URRACA, Auteur ; K. HOPE, Auteur ; A. K. VICTOR, Auteur ; T. G. BELGARD, Auteur ; R. MEMON, Auteur ; S. GOORHA, Auteur ; C. VALDEZ, Auteur ; Q. T. TRAN, Auteur ; S. SANCHEZ, Auteur ; J. RAMIREZ, Auteur ; M. DONALDSON, Auteur ; D. BRIDGES, Auteur ; L. T. REITER, Auteur . - 6p.
Langues : Anglais (eng)
in Molecular Autism > 9 (2018) . - 6p.
Mots-clés : Autism Genomic disorders Neurogenetic syndrome Stem cells mRNAseq Index. décimale : PER Périodiques Résumé : Background: The inability to analyze gene expression in living neurons from Angelman (AS) and Duplication 15q (Dup15q) syndrome subjects has limited our understanding of these disorders at the molecular level. Method: Here, we use dental pulp stem cells (DPSC) from AS deletion, 15q Duplication, and neurotypical control subjects for whole transcriptome analysis. We identified 20 genes unique to AS neurons, 120 genes unique to 15q duplication, and 3 shared transcripts that were differentially expressed in DPSC neurons vs controls. Results: Copy number correlated with gene expression for most genes across the 15q11.2-q13.1 critical region. Two thirds of the genes differentially expressed in 15q duplication neurons were downregulated compared to controls including several transcription factors, while in AS differential expression was restricted primarily to the 15q region. Here, we show significant downregulation of the transcription factors FOXO1 and HAND2 in neurons from 15q duplication, but not AS deletion subjects suggesting that disruptions in transcriptional regulation may be a driving factor in the autism phenotype in Dup15q syndrome. Downstream analysis revealed downregulation of the ASD associated genes EHPB2 and RORA, both genes with FOXO1 binding sites. Genes upregulated in either Dup15q cortex or idiopathic ASD cortex both overlapped significantly with the most upregulated genes in Dup15q DPSC-derived neurons. Conclusions: Finding a significant increase in both HERC2 and UBE3A in Dup15q neurons and significant decrease in these two genes in AS deletion neurons may explain differences between AS deletion class and UBE3A specific classes of AS mutation where HERC2 is expressed at normal levels. Also, we identified an enrichment for FOXO1-regulated transcripts in Dup15q neurons including ASD-associated genes EHPB2 and RORA indicating a possible connection between this syndromic form of ASD and idiopathic cases. En ligne : http://dx.doi.org/10.1186/s13229-018-0191-y Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=354 Transcriptional signatures of participant-derived neural progenitor cells and neurons implicate altered Wnt signaling in Phelan-McDermid syndrome and autism / Michael S. BREEN in Molecular Autism, 11 (2020)
[article]
Titre : Transcriptional signatures of participant-derived neural progenitor cells and neurons implicate altered Wnt signaling in Phelan-McDermid syndrome and autism Type de document : Texte imprimé et/ou numérique Auteurs : Michael S. BREEN, Auteur ; Andrew BROWNE, Auteur ; Gabriel E. HOFFMAN, Auteur ; Sofia STATHOPOULOS, Auteur ; Kristen BRENNAND, Auteur ; Joseph D. BUXBAUM, Auteur ; Elodie DRAPEAU, Auteur Article en page(s) : 53 p. Langues : Anglais (eng) Mots-clés : Autism spectrum disorder Neural progenitor cells Neurons RNA-sequencing Stem cells Index. décimale : PER Périodiques Résumé : BACKGROUND: Phelan-McDermid syndrome (PMS) is a rare genetic disorder with high risk of autism spectrum disorder (ASD), intellectual disability, and language delay, and is caused by 22q13.3 deletions or mutations in the SHANK3 gene. To date, the molecular and pathway changes resulting from SHANK3 haploinsufficiency in PMS remain poorly understood. Uncovering these mechanisms is critical for understanding pathobiology of PMS and, ultimately, for the development of new therapeutic interventions. METHODS: We developed human-induced pluripotent stem cell (hiPSC)-based models of PMS by reprogramming peripheral blood samples from individuals with PMS (n = 7) and their unaffected siblings (n = 6). For each participant, up to three hiPSC clones were generated and differentiated into induced neural progenitor cells (hiPSC-NPCs; n = 39) and induced forebrain neurons (hiPSC-neurons; n = 41). Genome-wide RNA-sequencing was applied to explore transcriptional differences between PMS probands and unaffected siblings. RESULTS: Transcriptome analyses identified 391 differentially expressed genes (DEGs) in hiPSC-NPCs and 82 DEGs in hiPSC-neurons, when comparing cells from PMS probands and unaffected siblings (FDR 5%). Genes under-expressed in PMS were implicated in Wnt signaling, embryonic development, and protein translation, while over-expressed genes were enriched for pre- and postsynaptic density genes, regulation of synaptic plasticity, and G-protein-gated potassium channel activity. Gene co-expression network analysis identified two modules in hiPSC-neurons that were over-expressed in PMS, implicating postsynaptic signaling and GDP binding, and both modules harbored a significant enrichment of genetic risk loci for developmental delay and intellectual disability. Finally, PMS-associated genes were integrated with other ASD hiPSC transcriptome findings and several points of convergence were identified, indicating altered Wnt signaling and extracellular matrix. LIMITATIONS: Given the rarity of the condition, we could not carry out experimental validation in independent biological samples. In addition, functional and morphological phenotypes caused by loss of SHANK3 were not characterized here. CONCLUSIONS: This is the largest human neural sample analyzed in PMS. Genome-wide RNA-sequencing in hiPSC-derived neural cells from individuals with PMS revealed both shared and distinct transcriptional signatures across hiPSC-NPCs and hiPSC-neurons, including many genes implicated in risk for ASD, as well as specific neurobiological pathways, including the Wnt pathway. En ligne : http://dx.doi.org/10.1186/s13229-020-00355-0 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=427
in Molecular Autism > 11 (2020) . - 53 p.[article] Transcriptional signatures of participant-derived neural progenitor cells and neurons implicate altered Wnt signaling in Phelan-McDermid syndrome and autism [Texte imprimé et/ou numérique] / Michael S. BREEN, Auteur ; Andrew BROWNE, Auteur ; Gabriel E. HOFFMAN, Auteur ; Sofia STATHOPOULOS, Auteur ; Kristen BRENNAND, Auteur ; Joseph D. BUXBAUM, Auteur ; Elodie DRAPEAU, Auteur . - 53 p.
Langues : Anglais (eng)
in Molecular Autism > 11 (2020) . - 53 p.
Mots-clés : Autism spectrum disorder Neural progenitor cells Neurons RNA-sequencing Stem cells Index. décimale : PER Périodiques Résumé : BACKGROUND: Phelan-McDermid syndrome (PMS) is a rare genetic disorder with high risk of autism spectrum disorder (ASD), intellectual disability, and language delay, and is caused by 22q13.3 deletions or mutations in the SHANK3 gene. To date, the molecular and pathway changes resulting from SHANK3 haploinsufficiency in PMS remain poorly understood. Uncovering these mechanisms is critical for understanding pathobiology of PMS and, ultimately, for the development of new therapeutic interventions. METHODS: We developed human-induced pluripotent stem cell (hiPSC)-based models of PMS by reprogramming peripheral blood samples from individuals with PMS (n = 7) and their unaffected siblings (n = 6). For each participant, up to three hiPSC clones were generated and differentiated into induced neural progenitor cells (hiPSC-NPCs; n = 39) and induced forebrain neurons (hiPSC-neurons; n = 41). Genome-wide RNA-sequencing was applied to explore transcriptional differences between PMS probands and unaffected siblings. RESULTS: Transcriptome analyses identified 391 differentially expressed genes (DEGs) in hiPSC-NPCs and 82 DEGs in hiPSC-neurons, when comparing cells from PMS probands and unaffected siblings (FDR 5%). Genes under-expressed in PMS were implicated in Wnt signaling, embryonic development, and protein translation, while over-expressed genes were enriched for pre- and postsynaptic density genes, regulation of synaptic plasticity, and G-protein-gated potassium channel activity. Gene co-expression network analysis identified two modules in hiPSC-neurons that were over-expressed in PMS, implicating postsynaptic signaling and GDP binding, and both modules harbored a significant enrichment of genetic risk loci for developmental delay and intellectual disability. Finally, PMS-associated genes were integrated with other ASD hiPSC transcriptome findings and several points of convergence were identified, indicating altered Wnt signaling and extracellular matrix. LIMITATIONS: Given the rarity of the condition, we could not carry out experimental validation in independent biological samples. In addition, functional and morphological phenotypes caused by loss of SHANK3 were not characterized here. CONCLUSIONS: This is the largest human neural sample analyzed in PMS. Genome-wide RNA-sequencing in hiPSC-derived neural cells from individuals with PMS revealed both shared and distinct transcriptional signatures across hiPSC-NPCs and hiPSC-neurons, including many genes implicated in risk for ASD, as well as specific neurobiological pathways, including the Wnt pathway. En ligne : http://dx.doi.org/10.1186/s13229-020-00355-0 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=427 Cell therapy approaches to autism: a review of clinical trial data / Jack PRICE in Molecular Autism, 11 (2020)
[article]
Titre : Cell therapy approaches to autism: a review of clinical trial data Type de document : Texte imprimé et/ou numérique Auteurs : Jack PRICE, Auteur Article en page(s) : 37 p. Langues : Anglais (eng) Mots-clés : Autism Cell therapy Clinical trials Stem cells Index. décimale : PER Périodiques Résumé : A number of clinical trials of cell therapies for autism spectrum disorder have been conducted, and some have published their outcomes. This review considers the data that have emerged from this small set of published trials, evaluates their success, and proposes further steps that could be taken if this field of endeavour is to be pursued further. A number of reservations arise from this tranche of studies, specifically the absence of identified therapeutic targets, and deficiencies in the therapeutic approach that is being employed. If this therapeutic direction is to be pursued further, then additional pre-clinical studies are recommended that might lead to improvements in patient stratification, biomarkers, the defined mode of action, and the preparation and identification of the therapeutic cells themselves. En ligne : http://dx.doi.org/10.1186/s13229-020-00348-z Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=427
in Molecular Autism > 11 (2020) . - 37 p.[article] Cell therapy approaches to autism: a review of clinical trial data [Texte imprimé et/ou numérique] / Jack PRICE, Auteur . - 37 p.
Langues : Anglais (eng)
in Molecular Autism > 11 (2020) . - 37 p.
Mots-clés : Autism Cell therapy Clinical trials Stem cells Index. décimale : PER Périodiques Résumé : A number of clinical trials of cell therapies for autism spectrum disorder have been conducted, and some have published their outcomes. This review considers the data that have emerged from this small set of published trials, evaluates their success, and proposes further steps that could be taken if this field of endeavour is to be pursued further. A number of reservations arise from this tranche of studies, specifically the absence of identified therapeutic targets, and deficiencies in the therapeutic approach that is being employed. If this therapeutic direction is to be pursued further, then additional pre-clinical studies are recommended that might lead to improvements in patient stratification, biomarkers, the defined mode of action, and the preparation and identification of the therapeutic cells themselves. En ligne : http://dx.doi.org/10.1186/s13229-020-00348-z Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=427 Distinct, dosage-sensitive requirements for the autism-associated factor CHD8 during cortical development / S. HURLEY in Molecular Autism, 12 (2021)
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