ArticleExperimental & molecular medicine2016
Concurrent progress of reprogramming and gene correction to overcome therapeutic limitation of mutant ALK2-iPSC.
Article in Experimental & molecular medicine, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
16 citing papers in PubMed, 24 citations in OpenAlex.
- Fibrodysplasia ossificans progressiva: genetic and clinical characterization in a cohort of Polish patients and review of potential therapies.Journal of applied genetics · 2026Review
- Review
- Gene Editing and Human iPSCs in Cardiovascular and Metabolic Diseases.Advances in experimental medicine and biology · 2023Article
- Simultaneous high-efficiency base editing and reprogramming of patient fibroblasts.Stem cell reports · 2021Article
- Episomal Induced Pluripotent Stem Cells: Functional and Potential Therapeutic Applications.Cell transplantation · 2019Review
- ACVR1 Function in Health and Disease.Cells · 2019Review
- Efficient exogenous DNA-free reprogramming with suicide gene vectors.Experimental & molecular medicine · 2019Article
- K120R mutation inactivates p53 by creating an aberrant splice site leading to nonsense-mediated mRNA decay.Oncogene · 2019Article
- Genome Editing in Induced Pluripotent Stem Cells using CRISPR/Cas9.Stem cell reviews and reports · 2018Review
- Generation of urine-derived induced pluripotent stem cells from a patient with phenylketonuria.Intractable & rare diseases research · 2018Article
- Application of human induced pluripotent stem cells to model fibrodysplasia ossificans progressiva.Bone · 2018Review
- AMPK downregulates ALK2 via increasing the interaction between Smurf1 and Smad6, leading to inhibition of osteogenic differentiation.Biochimica et biophysica acta. Molecular cell research · 2017Article
- Fibrodysplasia ossificans progressiva: Basic understanding and experimental models.Intractable & rare diseases research · 2017Review
- Therapeutic gene editing: delivery and regulatory perspectives.Acta pharmacologica Sinica · 2017Review
- Urine-derived induced pluripotent stem cells as a modeling tool to study rare human diseases.Intractable & rare diseases research · 2016Review
- Integrating Gene Correction in the Reprogramming and Transdifferentiation Processes: A One-Step Strategy to Overcome Stem Cell-Based Gene Therapy Limitations.Stem cells international · 2016Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Fibrodysplasia ossificans progressiva (FOP) syndrome is caused by mutation of the gene ACVR1, encoding a constitutive active bone morphogenetic protein type I receptor (also called ALK2) to induce heterotopic ossification in the patient. To genetically correct it, we attempted to generate the mutant ALK2-iPSCs (mALK2-iPSCs) from FOP-human dermal fibroblasts. However, the mALK2 leads to inhibitory pluripotency maintenance, or impaired clonogenic potential after single-cell dissociation as an inevitable step, which applies gene-correction tools to induced pluripotent stem cells (iPSCs). Thus, current iPSC-based gene therapy approach reveals a limitation that is not readily applicable to iPSCs with ALK2 mutation. Here we developed a simplified one-step procedure by simultaneously introducing reprogramming and gene-editing components into human fibroblasts derived from patient with FOP syndrome, and genetically treated it. The mixtures of reprogramming and gene-editing components are composed of reprogramming episomal vectors, CRISPR/Cas9-expressing vectors and single-stranded oligodeoxynucleotide harboring normal base to correct ALK2 c.617G>A. The one-step-mediated ALK2 gene-corrected iPSCs restored global gene expression pattern, as well as mineralization to the extent of normal iPSCs. This procedure not only helps save time, labor and costs but also opens up a new paradigm that is beyond the current application of gene-editing methodologies, which is hampered by inhibitory pluripotency-maintenance requirements, or vulnerability of single-cell-dissociated iPSCs.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.