지원사업
학술연구/단체지원/교육 등 연구자 활동을 지속하도록 DBpia가 지원하고 있어요.
커뮤니티
연구자들이 자신의 연구와 전문성을 널리 알리고, 새로운 협력의 기회를 만들 수 있는 네트워킹 공간이에요.
논문 기본 정보
- 자료유형
- 학술저널
- 저자정보
- 발행연도
- 2016.1
- 수록면
- 1 - 12 (12page)
이용수
초록· 키워드
오류제보하기
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.617G4A. 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.
목차
등록된 정보가 없습니다.
참고문헌 (0)
참고문헌 신청함께 읽어보면 좋을 논문
논문 유사도에 따라 DBpia 가 추천하는 논문입니다. 함께 보면 좋을 연관 논문을 확인해보세요!
-
Highly Efficient Reprogramming and Characterization of Induced Pluripotent Stem Cells by Using a Microwell Array
조직공학과 재생의학
2016 .01
-
Cellular Reprogramming and Its Application in Regenerative Medicine
조직공학과 재생의학
2015 .01
-
iPSC technology-Powerful hand for disease modeling and therapeutic screen
BMB Reports
2015 .01
-
Generation and characterization of integration-free induced pluripotent stem cells from patients with autoimmune disease
Experimental and Molecular Medicine
2016 .01
-
Characterization and Bioreactor Expansion of iPSCs for the Treatment of Osteoarthritis
한국생물공학회 학술대회
2024 .09
-
Characterization of myocardial cells derived from inducible pluripotent stem cells(iPSCs)
한국생물공학회 학술대회
2018 .04
-
Fast colony formation during human iPSC generation using magnetic nanoparticle
한국생물공학회 학술대회
2016 .04
-
Study on Quality Control Markers of Cardiomyocytes Derived from Human Inducible Pluripotent Stem Cells (iPSCs)
한국생물공학회 학술대회
2019 .04
-
Efficient exogenous DNA-free reprogramming with suicide gene vectors
Experimental and Molecular Medicine
2019 .01
-
Small-molecule-mediated reprogramming: a silver lining for regenerative medicine
Experimental and Molecular Medicine
2020 .02
-
Advances and challenges in modeling inherited peripheral neuropathies using iPSCs
Experimental and Molecular Medicine
2024 .06
-
Rapid Non-Invasive Cell Type Identification for iPSC Cell Therapy with Artificial Intelligence-based Holotomography Analysis
한국생물공학회 학술대회
2022 .09
-
Efficient Reprogramming of Human Somatic Cells into Induced Pluripotent Stem Cells by Intracellular Delivery of RNA Nanoparticle
한국생물공학회 학술대회
2020 .10
-
Development of High-throughput Versatile iPSC-based Organoid Platform Using 3D Bioprinting
한국고분자학회 학술대회 연구논문 초록집
2023 .04
-
Enhanced Reprogramming Efficiency of Induced Pluripotent Stem Cells (iPSCs) Using Lin28-30Kc19 Fusion Protein
한국생물공학회 학술대회
2016 .04
-
Extracellular vesicles from pluripotent stem cell-derived mesenchymal stem cells acquire a stromal modulatory proteomic pattern during differentiation
Experimental and Molecular Medicine
2018 .01
-
Comparison of different culture conditions for human iPSC and MSC
한국생물공학회 학술대회
2018 .04
-
Separating various pluripotent state of iPSCs via magnetization of nanoclusters magnetophoresis devices
한국고분자학회 학술대회 연구논문 초록집
2017 .04
-
Mitochondrial genome mutations in mesenchymal stem cells derived from human dental induced pluripotent stem cells
BMB Reports
2019 .01
이 논문의 저자 정보
최근 본 자료
댓글(0)
0