Abstract:
:The Global Alliance for iPSC Therapies (GAiT) is a new initiative to support the implementation and clinical application of therapies derived from pluripotent stem cells to the benefit of patients globally. GAiT's mission is to serve as a central, international resource for those organisations developing therapies from clinical-grade induced pluripotent stem cells, and to support the expansion of this nascent field. With the support of its international partners, GAiT already has an early position on manufacturing, regulatory and quality standards. This article details GAiT's development, its mission and structure, as well as how, and by whom, it is funded. The article ends with brief overview of current and upcoming activities.
journal_name
Stem Cell Resjournal_title
Stem cell researchauthors
Sullivan S,Ginty P,McMahon S,May M,Solomon SL,Kurtz A,Stacey GN,Bennaceur Griscelli A,Li RA,Barry J,Song J,Turner MLdoi
10.1016/j.scr.2020.102036subject
Has Abstractpub_date
2020-12-01 00:00:00pages
102036eissn
1873-5061issn
1876-7753pii
S1873-5061(20)30337-8journal_volume
49pub_type
杂志文章abstract::Large-scale production of human induced pluripotent stem cells (hiPSCs) by robust and economic methods has been one of the major challenges for translational realization of hiPSC technology. Here we demonstrate a scalable culture system for hiPSC expansion using the E8 chemically defined and xeno-free medium under eit...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2013.07.011
更新日期:2013-11-01 00:00:00
abstract::Two distinct types of embryonic pluripotent stem cells can be established from either the inner cell mass (ICM) of preimplantation blastocyst (leukemia inhibitory factor (LIF)-dependent embryonic stem cell, ESC, called naive state) or the epiblast of postimplantation fetuses (fibroblast growth factor 2 (FGF2)-dependen...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2014.04.012
更新日期:2014-07-01 00:00:00
abstract::Human iPSC line N44SV.5 was generated from primary normal human dermal fibroblasts belonging to the European mitochondrial haplogroup U. For this purpose, reprogramming factors Oct3/4, Sox2, Klf4, and cMyc were delivered using a non-integrative methodology that involves the use of Sendai virus. ...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2015.12.010
更新日期:2016-01-01 00:00:00
abstract::The KCL034 human embryonic stem cell line was derived from a normal healthy blastocyst donated for research. The ICM was isolated using laser microsurgery and plated on γ-irradiated human foreskin fibroblasts. Both the derivation and cell line propagation were performed in an animal product-free environment and under ...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2015.12.034
更新日期:2016-01-01 00:00:00
abstract::Salisphere-derived adult epithelial cells have been used to improve saliva production of irradiated mouse salivary glands. Importantly, optimization of the cellular composition of salispheres could improve their regenerative capabilities. The Rho Kinase (ROCK) inhibitor, Y27632, has been used to increase the prolifera...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2019.101608
更新日期:2019-12-01 00:00:00
abstract::Chimeric animals are made up of cells from two separate zygotes. Human/non-human animal chimeras have been used for a number of research purposes, including human disease modeling. Pluripotent stem cell (PSC) research has relied upon the chimera approach to examine the developmental potential of stem cells, to determi...
journal_title:Stem cell research
pub_type: 杂志文章,评审
doi:10.1016/j.scr.2017.09.005
更新日期:2017-10-01 00:00:00
abstract::The human transcription factor NKX2-5 plays an important role in cardiac formation and development, and thus it can be used for isolation of cardiomyocytes (CMs) differentiated from human pluripotent stem cells (hPSCs). Here, we knocked-in enhanced GFP (eGFP) and Pac (a puromycin resistant gene; PuroR) into the exon 1...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2020.102120
更新日期:2020-12-10 00:00:00
abstract::Liver cirrhosis accompanied with hepatic encephalopathy commonly causes cognitive impairment in patients. To model this disease, two independent patient specific induced pluripotent stem cell-line (iPSC) clones, NCCSi011-A and NCCSi011-B were generated by reprogramming the CD4+ T cells of an Indian male patient suffer...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2020.101992
更新日期:2020-10-01 00:00:00
abstract::The KCL018 human embryonic stem cell line was derived from an embryo donated for research that carried an autosomal dominant mutation affecting one allele of the DMPK gene encoding the dystrophia myotonica protein kinase (2200 trinucleotide repeats; 14 for the normal allele). The ICM was isolated using laser microsurg...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2016.01.004
更新日期:2016-03-01 00:00:00
abstract::Members of the Fanconi anemia (FA) protein family are involved in multiple cellular processes including response to DNA damage and oxidative stress. Here we show that a major FA protein, Fancd2, plays a role in mitochondrial biosynthesis through regulation of mitochondrial translation. Fancd2 interacts with Atad3 and ...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2019.101550
更新日期:2019-10-01 00:00:00
abstract::Human embryonic stem cell (hESC) line chHES-478 was derived from abnormal blastocyst diagnosed with albinism after preimplantation genetic diagnosis (PGD) treatment. DNA sequencing analysis confirmed that chHES-478 cell line carried a compound heterozygous mutation, c.896G>A(p.Arg299His) and c.929_930insC(p.Pro310Glnf...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2016.11.004
更新日期:2016-11-01 00:00:00
abstract::Dominant deafness-onychodystrophy (DDOD) syndrome is a rare, autosomal dominant inherited disorder with no concrete therapies in human. We previously identified c.1516 C > T (p.Arg506*) in ATP6V1B2 as cause of DDOD syndrome, accounting for all cases of this genetic disorder. The induced pluripotent stem cell (iPSC) li...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2020.101986
更新日期:2020-10-01 00:00:00
abstract::Mesenchymal stem cells (MSC) have been isolated from almost every adult tissue. In cord blood (CB), different non-hematopoietic CD45-, CD34- adherent cell populations can be generated: the cord blood derived MSC (CB-MSC), that behave almost like MSC from bone marrow (BM-MSC), and unrestricted somatic stem cells (USSC)...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2010.03.004
更新日期:2010-07-01 00:00:00
abstract::Among the known causative genes of familial ALS, SOD1mutation is one of the most common. It encodes for the ubiquitous detoxifying copper/zinc binding SOD1 enzyme, whose mutations selectively cause motor neuron death, although the mechanisms are not as yet clear. What is known is that mutant-mediated toxicity is not c...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2020.101924
更新日期:2020-07-25 00:00:00
abstract::Stem cells can differentiate into various body tissues and organs and thus are considered as promising tools for cell therapy and tissue engineering. Early passage stem cells have high differentiation ability compared to late passage stem cells. Thus, it is important to use early passage stem cells in cell therapy. He...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2019.101475
更新日期:2019-07-01 00:00:00
abstract::GADD45A is a DNA damage and stressful growth arrest inducible protein, also it is shown to a be tumor suppressor gene and a chromatin relaxer associated with opening chromatin during the somatic reprogramming. However, its role in human embryonic stem cells and human embryonic stem cell modeled development has been me...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2020.102090
更新日期:2020-12-01 00:00:00
abstract::This study elucidated the stage-specific roles of FGF2 signaling during neural development using in-vitro human embryonic stem cell-based developmental modeling. We found that the dysregulation of FGF2 signaling prior to the onset of neural induction resulted in the malformation of neural rosettes (a neural tube-like ...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2016.08.012
更新日期:2016-09-01 00:00:00
abstract::Pathological myopia (PM) is a retinal degenerative disease with an increasing prevalence in Asia. The peripheral blood mononuclear cells (PBMCs) from a patient with PM were successfully reprogrammed to induced pluripotent stem cells (iPSCs) using integration-free method, Sendai viral (SeV) vectors expressing OCT4, SOX...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2018.101369
更新日期:2019-01-01 00:00:00
abstract::Expanded human T cells from a Japanese female with recessive dystrophic epidermolysis bullosa (RDBE) were used to generate integration-free induced pluripotent stem cells (iPSCs) by exogenous expression of four reprogramming factors, OCT3/4, SOX2, cMYC, KLF4, using Sendai virus vector (SeVdp). The authenticity of esta...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2016.05.003
更新日期:2016-07-01 00:00:00
abstract::Generating human podocytes in vitro could offer a unique opportunity to study human diseases. Here, we describe a simple and efficient protocol for obtaining functional podocytes in vitro from human induced pluripotent stem cells. Cells were exposed to a three-step protocol, which induced their differentiation into in...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2016.06.001
更新日期:2016-07-01 00:00:00
abstract::We generated an induced human pluripotent stem cell line from a child with microcephaly carrying compound heterozygous mutations of TYW1 inherited from healthy parents. This iPS cell line showed typical embryonic stem cell-like morphology, expressed pluripotent markers that comparable to human embryonic stem cells. Mo...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2020.101783
更新日期:2020-05-01 00:00:00
abstract::Induced pluripotent stem cells (iPSCs) were generated from peripheral blood mononuclear cells (PBMCs) obtained from a 60-year-old female diagnosed with sporadic amyotrophic lateral sclerosis (sALS). The iPSCs shared the same karyotype with the parent PBMCs, expressed pluripotency stem cell markers, and demonstrated tr...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2020.101841
更新日期:2020-05-01 00:00:00
abstract::CHCHD2 mutation has been reported as a potential cause of a rare form of familial Parkinson's disease. Recently, a novel CHCHD2 mutation was identified in a family with Parkinson's disease. The dermal fibroblasts of the patient were obtained and successfully transformed into induced pluripotent stem cells(iPSCs), empl...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2018.08.011
更新日期:2018-10-01 00:00:00
abstract::FOS is component of the AP-1 complex and has been reported to be involved in many cellular functions, including cell proliferation, differentiation, survival, angiogenesis, hematopoiesis and cancer progress. To further understand the exact role of FOS in these processes, here we created two FOS knockout human embryoni...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2019.101479
更新日期:2019-08-01 00:00:00
abstract::Leucine rich repeat kinase (LRRK2) is the most prevalent genetic cause for Parkinson's disease. LRRK2 p.G2385R is an Asian specific genetic risk factor for sporadic Parkinson's disease. We generated two induced pluripotent stem cells (iPSCs), IBMS-iPSC-018-09 and IBMS-iPSC-020-01, from the peripheral blood mononuclear...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2018.01.034
更新日期:2018-04-01 00:00:00
abstract::An important practical limitation of the three-dimensional geometry of stem-cell derived intestinal organoids is that it prevents easy access to the apical epithelium for testing food components, microorganisms, bioactive and toxic compounds. To this end, we here report on a new robust method for generating confluent ...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2018.02.013
更新日期:2018-04-01 00:00:00
abstract::Human epicardium-derived cells (EPDC) were reprogrammed to generate two iPSC lines, MCDU1i-EPDC and MCDU2i-EPDC, by nucleofection of episomal-based plasmids expressing the reprogramming factors OCT4, SOX2, KLF4, c-MYC, NANOG and LIN28. Pluripotency was confirmed in vitro by immunofluorescence analysis and embryoid bod...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2017.02.007
更新日期:2017-04-01 00:00:00
abstract::Maple syrup urine disease type Ib (MSUD Ib) is an autosomal recessive genetic metabolic disease caused by homozygous or compound heterozygous mutation in BCKDHB on chromosome 6q14. We generated an induced pluripotent stem cell (iPSC) line from peripheral blood mononuclear cells (PBMCs) of a 5-day-old boy with MSUD Ib ...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2019.101579
更新日期:2019-10-01 00:00:00
abstract::We studied the susceptibility of human embryonic stem cells and derived contractile embryoid bodies from WAO9, HUES-5 and HUES-16 cell lines to Coxsackievirus B infection. After validating stem cell-like properties and cardiac phenotype, Coxsackievirus B receptors CAR and DAF, as well as type I interferon receptors we...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2010.09.002
更新日期:2011-01-01 00:00:00
abstract::Autosomal recessive Stargardt disease is the most common cause of inherited retinal disease. In this report, we describe the generation and characterization of two human induced pluripotent stem cell (iPSC) lines from a patient with compound heterozygous mutations in the ABCA4 gene (c.[768G>T];[6079C>T]). Patient derm...
journal_title:Stem cell research
pub_type: 杂志文章
doi:10.1016/j.scr.2020.101947
更新日期:2020-10-01 00:00:00