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Utilization avenues for induced pluripotent stem cells in autism

Yıl 2019, Cilt: 41 Sayı: 1, 115 - 120, 28.03.2019

Ceymi Doenyas , Nelisa Türkoğlu Laçin

https://doi.org/10.7197/223.vi.543431

Öz

Objective: Autism spectrum disorder
(ASD) is a neurodevelopmental difference with increasing global prevalence.
Yet, the etiology of ASD is still not unraveled. Individuals with ASD
experience difficulties with social skills and communication, and exhibit
repetitive and restrictive interests and behaviors.



Method: There is yet no
comprehensive model of autism that can explain all aspects of autism.
Similarly, there exists no known treatment for it. Induced pluripotent stem
cell (iPSC), which has been widely used in the regenerative treatment of many
diseases in recent years, is promising for a better understanding of the causes
of autism.



Results: Autism modelling with
iPSCs provides several opportunities for understanding the mechanisms
underlying autism and developing personalized treatments.



Conclusions: This review
examines the use of iPSCs in ASD to date, their advantages, and the findings
obtained via iPSC modeling of autism.

Anahtar Kelimeler

autism induced pluripotent stem cell modelling

Kaynakça

  • REFERENCES1- American Psychiatric Association. Diagnostic and statistical manual of mental disorders (DSM-5®). American Psychiatric Pub, 2013. 2- Baio J, Wiggins L, Christensen DL, Maenner MJ, Daniels J, Warren Z… Durkin MS. Prevalence of autism spectrum disorder among children aged 8 years—Autism and Developmental Disabilities Monitoring Network, 11 Sites, United States, 2014. MMWR Surveill Summ 2018; 67(6): 1-23.3- Karaşahin T. Embriyonik Kök Hücreler. Erciyes Üniv Vet Fak Derg 2012; 9(1):65-71.4- İskender B, Canatan H. Uyarılmış pluripotent Kök Hücreler ve Hücre Tedavisi. JCEI 2013; 4(4): 550-561.5- Avcılar H, Saraymen B, Özturan OÖ, Köker MY. Embriyonik Kök Hücreler ve Uyarılmış Pluripotent Kök Hücreler. Asthma Allergy Immunol 2017; 15:1-6.6- Ateş U. Kök Hücreyi Tanıyalım. FNG & Bilim Tıp Transplantasyon Dergisi 2016; 1(1):19-28.7- Takahashi K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, Tomoda K, Yamanaka S. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007; 131(5): 861-872.8- Kimbrel EA & Lanza R. Current status of pluripotent stem cells: moving the first therapies to the clinic. Nat Rev Drug Discov 2015; 14(10): 681-692.9- Liu GH, Ding Z, Belmonte JCI. iPSC technology to study human aging and aging-related disorders. Current Opin Cell Biol 2012; 24(6): 765-774.10- Paşca SP, Portmann T, Voineagu I, Yazawa M, Shcheglovitov A, Paşca AM… Bernstein JA. Using iPSC-derived neurons to uncover cellular phenotypes associated with Timothy syndrome. Nat Med 2011; 17(12):1657-1662.11- Habela CW, Song H, Ming GL. Modeling synaptogenesis in schizophrenia and autism using human iPSC derived neurons. Moll Cell Neurosci 2016; 73: 52-62.12- Vitrac A, Cloëz-Tayarani I. Induced pluripotent stem cells as a tool to study brain circuits in autism-related disorders. Stem Cell Res Ther 2018; 9(1): 226.13- Acab A, Muotri AR. The use of induced pluripotent stem cell technology to advance autism research and treatment. Neurotherapeutics 2015; 12(3), 534-545.14- Malkova NV, Collin ZY, Hsiao EY, Moore MJ, Patterson PH. Maternal immune activation yields offspring displaying mouse versions of the three core symptoms of autism. Brain Behav Immun 2012;26(4):607-16.15- McFarlane HG, Kusek GK, Yang M, Phoenix JL, Bolivar VJ, Crawley JN. Autism‐like behavioral phenotypes in BTBR T+ tf/J mice. Genes Brain Behav 2008;7(2):152-63.16- Connacher RJ, DiCicco-Bloom E, Millonig JH. Using Human Induced Neural Precursor Cells to Define Early Neurodevelopmental Defects in Syndromic and Idiopathic Autism. Current Pharmacology Reports 2018;4(6): 422-35.17- Cocks G, Curran S, Gami P, Uwanogho D, Jeffries AR, Kathuria A, Steckler T, et al. The utility of patient specific induced pluripotent stem cells for the modelling of Autistic Spectrum Disorders. Psychopharmacology 2014;231(6): 1079-88.18- Nestor MW, Phillips AW, Artimovich E, Nestor JE, Hussman JP, Blatt GJ. Human inducible pluripotent stem cells and autism spectrum disorder: Emerging technologies. Autism Res 2016; 9(5): 513-35.19- Marchetto MC, Carromeu C, Acab A, Yu D, Yeo GW, Mu Y, Muotri AR, et al. A model for neural development and treatment of Rett syndrome using human induced pluripotent stem cells. Cell 2010;143(4): 527-39.20- St. Clair D, Johnstone M. Using mouse transgenic and human stem cell technologies to model genetic mutations associated with schizophrenia and autism. Philos Trans R Soc Lond B Biol Sci 2018; 373(1742): 20170037.21- Darville H, Poulet, A, Rodet-Amsellem F, Chatrousse L, Pernelle J, Boissart C, Cogé, F, et al. Human pluripotent stem cell-derived cortical neurons for high throughput medication screening in autism: a proof of concept study in SHANK3 haploinsufficiency syndrome. EBioMedicine 2016; 9: 293-305.22- Freitas BC, Trujillo CA, Carromeu C, Yusupova M, Herai RH, Muotri AR. Stem cells and modeling of autism spectrum disorders. Exp Neurol 2014; 260: 33-43.23- Russo FB, Freitas BC, Pignatari GC, Fernandes IR, Sebat J, Muotri AR, Beltrão-Braga PCB. Modeling the interplay between neurons and astrocytes in autism using human induced pluripotent stem cells. Biol Psychiatry 2018; 83(7): 569-78.

Uyarılmış pluripotent kök hücrelerin otizm’de kullanım alanları

Yıl 2019, Cilt: 41 Sayı: 1, 115 - 120, 28.03.2019

Ceymi Doenyas , Nelisa Türkoğlu Laçin

https://doi.org/10.7197/223.vi.543431

Öz

Amaç: Otizm
spektrum bozukluğu, dünya çapında yaygınlığı giderek artan fakat sebepleri hala
tamamen bilinmeyen, nörogelişimsel bir farklılıktır. Otizmli bireyler, sosyal
beceri ve iletişim zorlukları yaşarlar; tekrar eden ve kısıtlayıcı ilgi
alanları ile davranışlar gösterirler. Otizmin bilinen bir tedavisi veya otizmin
bütün unsurlarını açıklayabilen kapsamlı bir model henüz yoktur.

Yöntem: Son
yıllarda birçok hastalığın rejeneratif tedavisinde oldukça yaygın olarak
kullanılan kök hücrelerden, uyarılmış pluripotent kök hücre (UPKH), otizmin
sebeplerinin daha iyi anlaşılması için umut vaat edicidir.

Bulgular: UPKH’ler
ile yürütülen otizm modellemeleri, otizme neden olan mekanizmaların
anlaşılması, bozuklukların düzeltilmesi ve kişiye özel tedavilerin
geliştirilmesi için çeşitli fırsatlar sunmaktadır.











Sonuç:  Bu derleme, UPKH’lerin şu ana kadar otizmdeki
kullanım alanlarını, avantajlarını ve UPKH modellemeleri ile elde edilen
bulguları incelemektedir.

Anahtar Kelimeler

otizm uyarılmış pluripotent kök hücre modelleme

Kaynakça

  • REFERENCES1- American Psychiatric Association. Diagnostic and statistical manual of mental disorders (DSM-5®). American Psychiatric Pub, 2013. 2- Baio J, Wiggins L, Christensen DL, Maenner MJ, Daniels J, Warren Z… Durkin MS. Prevalence of autism spectrum disorder among children aged 8 years—Autism and Developmental Disabilities Monitoring Network, 11 Sites, United States, 2014. MMWR Surveill Summ 2018; 67(6): 1-23.3- Karaşahin T. Embriyonik Kök Hücreler. Erciyes Üniv Vet Fak Derg 2012; 9(1):65-71.4- İskender B, Canatan H. Uyarılmış pluripotent Kök Hücreler ve Hücre Tedavisi. JCEI 2013; 4(4): 550-561.5- Avcılar H, Saraymen B, Özturan OÖ, Köker MY. Embriyonik Kök Hücreler ve Uyarılmış Pluripotent Kök Hücreler. Asthma Allergy Immunol 2017; 15:1-6.6- Ateş U. Kök Hücreyi Tanıyalım. FNG & Bilim Tıp Transplantasyon Dergisi 2016; 1(1):19-28.7- Takahashi K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, Tomoda K, Yamanaka S. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007; 131(5): 861-872.8- Kimbrel EA & Lanza R. Current status of pluripotent stem cells: moving the first therapies to the clinic. Nat Rev Drug Discov 2015; 14(10): 681-692.9- Liu GH, Ding Z, Belmonte JCI. iPSC technology to study human aging and aging-related disorders. Current Opin Cell Biol 2012; 24(6): 765-774.10- Paşca SP, Portmann T, Voineagu I, Yazawa M, Shcheglovitov A, Paşca AM… Bernstein JA. Using iPSC-derived neurons to uncover cellular phenotypes associated with Timothy syndrome. Nat Med 2011; 17(12):1657-1662.11- Habela CW, Song H, Ming GL. Modeling synaptogenesis in schizophrenia and autism using human iPSC derived neurons. Moll Cell Neurosci 2016; 73: 52-62.12- Vitrac A, Cloëz-Tayarani I. Induced pluripotent stem cells as a tool to study brain circuits in autism-related disorders. Stem Cell Res Ther 2018; 9(1): 226.13- Acab A, Muotri AR. The use of induced pluripotent stem cell technology to advance autism research and treatment. Neurotherapeutics 2015; 12(3), 534-545.14- Malkova NV, Collin ZY, Hsiao EY, Moore MJ, Patterson PH. Maternal immune activation yields offspring displaying mouse versions of the three core symptoms of autism. Brain Behav Immun 2012;26(4):607-16.15- McFarlane HG, Kusek GK, Yang M, Phoenix JL, Bolivar VJ, Crawley JN. Autism‐like behavioral phenotypes in BTBR T+ tf/J mice. Genes Brain Behav 2008;7(2):152-63.16- Connacher RJ, DiCicco-Bloom E, Millonig JH. Using Human Induced Neural Precursor Cells to Define Early Neurodevelopmental Defects in Syndromic and Idiopathic Autism. Current Pharmacology Reports 2018;4(6): 422-35.17- Cocks G, Curran S, Gami P, Uwanogho D, Jeffries AR, Kathuria A, Steckler T, et al. The utility of patient specific induced pluripotent stem cells for the modelling of Autistic Spectrum Disorders. Psychopharmacology 2014;231(6): 1079-88.18- Nestor MW, Phillips AW, Artimovich E, Nestor JE, Hussman JP, Blatt GJ. Human inducible pluripotent stem cells and autism spectrum disorder: Emerging technologies. Autism Res 2016; 9(5): 513-35.19- Marchetto MC, Carromeu C, Acab A, Yu D, Yeo GW, Mu Y, Muotri AR, et al. A model for neural development and treatment of Rett syndrome using human induced pluripotent stem cells. Cell 2010;143(4): 527-39.20- St. Clair D, Johnstone M. Using mouse transgenic and human stem cell technologies to model genetic mutations associated with schizophrenia and autism. Philos Trans R Soc Lond B Biol Sci 2018; 373(1742): 20170037.21- Darville H, Poulet, A, Rodet-Amsellem F, Chatrousse L, Pernelle J, Boissart C, Cogé, F, et al. Human pluripotent stem cell-derived cortical neurons for high throughput medication screening in autism: a proof of concept study in SHANK3 haploinsufficiency syndrome. EBioMedicine 2016; 9: 293-305.22- Freitas BC, Trujillo CA, Carromeu C, Yusupova M, Herai RH, Muotri AR. Stem cells and modeling of autism spectrum disorders. Exp Neurol 2014; 260: 33-43.23- Russo FB, Freitas BC, Pignatari GC, Fernandes IR, Sebat J, Muotri AR, Beltrão-Braga PCB. Modeling the interplay between neurons and astrocytes in autism using human induced pluripotent stem cells. Biol Psychiatry 2018; 83(7): 569-78.
Toplam 1 adet kaynakça vardır.

Ayrıntılar

Birincil Dil İngilizce
Konular Sağlık Kurumları Yönetimi
Bölüm Dahili Tıp Bilimleri Araştırma Yazıları
Yazarlar

Ceymi Doenyas

Nelisa Türkoğlu Laçin

Yayımlanma Tarihi 28 Mart 2019
Kabul Tarihi 27 Mart 2019
Yayımlandığı Sayı Yıl 2019 Cilt: 41 Sayı: 1

Kaynak Göster

AMA Doenyas C, Türkoğlu Laçin N. Utilization avenues for induced pluripotent stem cells in autism. CMJ. Mart 2019;41(1):115-120. doi:10.7197/223.vi.543431