iPSC-based drug screening for Huntington׳s disease

Zhang, Ningzhe; Bailus, Barbara J.; Ring, Karen; Ellerby, Lisa M.

doi:10.1016/j.brainres.2015.09.020

Cited by 33 publications

(23 citation statements)

References 134 publications

(124 reference statements)

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“…Moreover, pre-clinical safety and efficacy tests could be performed on patient-derived iPSCs, control iPSCs, and corrected iPSCs from patients to find potential side-effects. In that sense, the generation of isogenic iPSC lines with the correction of the disease-causing mutation could solve the problems related to the genetic background variance [25]. In 2018, Japanese researchers revealed ropinirole hydrochloride as a potential candidate drug for ALS treatment.…”

Section: The New Age Of Drug Discoverymentioning

confidence: 99%

The Challenge of Bringing iPSCs to the Patient

Ortuño-Costela

Cerrada

García-López

et al. 2019

IJMS

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The implementation of induced pluripotent stem cells (iPSCs) in biomedical research more than a decade ago, resulted in a huge leap forward in the highly promising area of personalized medicine. Nowadays, we are even closer to the patient than ever. To date, there are multiple examples of iPSCs applications in clinical trials and drug screening. However, there are still many obstacles to overcome. In this review, we will focus our attention on the advantages of implementing induced pluripotent stem cells technology into the clinics but also commenting on all the current drawbacks that could hinder this promising path towards the patient.In this review, we summarize the main applications of iPSCs in the clinics, the progress achieved to date, and the path towards the patient, including the possible drawbacks that might appear in this process.

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Section: The New Age Of Drug Discoverymentioning

confidence: 99%

The Challenge of Bringing iPSCs to the Patient

Ortuño-Costela

Cerrada

García-López

et al. 2019

IJMS

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“…Thus, the drug screening results are more applicable in vivo. As an example, hiPSC-based drug screening for Huntington's disease has been established [97] developed. The applications of hiPSCs that have been reprogramed from patients of heritable, genetic diseases has been summarized by Wonhee Suh in a review paper [98].…”

Section: Drug Screeningmentioning

confidence: 99%

hiPSC-Based Tissue Organoid Regeneration

Qu¹,

Yucer²,

Garcia³

et al. 2018

Tissue Regeneration

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Induced pluripotent stem cells (iPSCs) are generated from terminally differentiated cells and have the potential to differentiate to any organs originated from the embryonic germ layers. Extensive effort has been made to establish protocols for direct in vitro conversion of human iPSCs (hiPSCs) to different cell types/organs. Importantly, hiPSCs can be generated from patients with known genetic mutations that predispose to high-risks of specific disease development. Thus, the hiPSCs technology provides unlimited resources for creating patient-specific disease models. hiPSC-derived three-dimensional "organoid" models have recently emerged as a powerful tool to recapitulate the physiologically-relevant process of disease progression in vitro. In this chapter, we will discuss the current advancement of organoid regeneration from hiPSCs and the applications of hiPSCs-derived organoids. The limitations and challenges of this approach will also be discussed here.

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“…Therefore, it is not surprising that modeling autism through generation of iPSCs is currently an emerging topic. Potential advantages and disadvantages of using iPSCs in other neurodevelopmental research and in ASD are widely discussed and reviewed in (Okano & Yamanaka 2014;Srikanth & Young-Pearse 2014;Haston & Finkbeiner 2015;Payne et al 2015;Sullivan & Young-Pearse 2015;Wan et al 2015;Zhang et al 2015).…”

Section: Autistic Networkmentioning

confidence: 99%

Modeling the autistic cell: iPSCs recapitulate developmental principles of syndromic and nonsyndromic ASD

Ben-Reuven

Reiner

2016

Dev Growth Differ

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The opportunity to model autism spectrum disorders (ASD) through generation of patient-derived induced pluripotent stem cells (iPSCs) is currently an emerging topic. Wide-scale research of altered brain circuits in syndromic ASD, including Rett Syndrome, Fragile X Syndrome, Angelman's Syndrome and sporadic Schizophrenia, was made possible through animal models. However, possibly due to species differences, and to the possible contribution of epigenetics in the pathophysiology of these diseases, animal models fail to recapitulate many aspects of ASD. With the advent of iPSCs technology, 3D cultures of patient-derived cells are being used to study complex neuronal phenotypes, including both syndromic and nonsyndromic ASD. Here, we review recent advances in using iPSCs to study various aspects of the ASD neuropathology, with emphasis on the efforts to create in vitro model systems for syndromic and nonsyndromic ASD. We summarize the main cellular activity phenotypes and aberrant genetic interaction networks that were found in iPSC-derived neurons of syndromic and nonsyndromic autistic patients.

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iPSC-based drug screening for Huntington׳s disease

Cited by 33 publications

References 134 publications

The Challenge of Bringing iPSCs to the Patient

The Challenge of Bringing iPSCs to the Patient

hiPSC-Based Tissue Organoid Regeneration

Modeling the autistic cell: iPSCs recapitulate developmental principles of syndromic and nonsyndromic ASD

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