Cited by 213Pluripotent Stem Cells ResearchSkin and Cellular Biology ResearchCRISPR and Genetic Engineering

The DOI matches its Crossref record; 39 of the 39 references it cites were found in a registry or an index.

Human COL7A1 -corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa

Open access

Sebastiano, Vittorio, Zhen, Hanson Hui, Haddad, Bahareh, Bashkirova, Elizaveta, Melo, Sandra P., Wang, Pei, Leung, Thomas L., Siprashvili, Zurab, Tichy, Andrea, Li, Jiang, Ameen, Mohammed, Hawkins, John, Lee, Susie, Li, Lingjie, Schwertschkow, Aaron, Bauer, Gerhard, Lisowski, Leszek, Kay, Mark A., Kim, Seung K., Lane, Alfred T., Wernig, Marius, Oro, Anthony E.·2014·Science Translational Medicine

DOI 10.1126/scitranslmed.3009540PMC PMC4428910

Open this paper in CoChat — free account

Abstract

Patient-specific iPSCs were derived, corrected, and differentiated into human skin grafts for the treatment of recessive dystrophic epidermolysis bullosa.

The claims and statistics of this paper haven’t been checked yet.

Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.

Integrity signals

Registry

Crossref · 10.1126/scitranslmed.3009540

Cites

39 of 39 references verified0 citation errors · 0 not checkable

Statistics

Claims the paper makes

The claims and statistics of this paper haven’t been checked yet.

Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.

Claims other papers make about this one

Each is checked against this paper’s own text: does it say what the citing paper says it does?

Self-check

Percentages, sums and reported differences re-computed from the paper’s own numbers.

The self-check needs the paper’s full text.

The Sequence Alignment/Map format and SAMtools
Li, H., Handsaker, B., Wysoker, A., Fennell, T., Ruan, J., Homer, N., Marth, G., Abecasis, G., Durbin, R., & 1000 Genome Project Data Processing Subgroup (2009) · Bioinformatics · doi:10.1093/bioinformatics/btp352 · Cited by 62,041
Fast and accurate short read alignment with Burrows-Wheeler transform
Li, H., & Durbin, R. (2009) · Bioinformatics · doi:10.1093/bioinformatics/btp324 · Cited by 58,607
Systematic and integrative analysis of large gene lists using DAVID bioinformatics resources
Huang, D. W., Sherman, B. T., & Lempicki, R. A. (2009) · Nature Protocols · doi:10.1038/nprot.2008.211 · Cited by 35,890
ANNOVAR: functional annotation of genetic variants from high-throughput sequencing data
Wang, K., Li, M., & Hakonarson, H. H. (2010) · Nucleic Acids Research · doi:10.1093/nar/gkq603 · Cited by 14,724
Inherited epidermolysis bullosa: Updated recommendations on diagnosis and classification
Fine, J., Bruckner‐Tuderman, L., Eady, R. A. J., Bauer, E. A., Bauer, J. W., Has, C., Heagerty, A., Hintner, H., Hovnanian, A., Jonkman, M. F., et al. (2014) · Journal of the American Academy of Dermatology · doi:10.1016/j.jaad.2014.01.903 · Cited by 934
Induced Pluripotent Stem Cell Generation Using a Single Lentiviral Stem Cell Cassette
Sommer, C. A., Stadtfeld, M., Murphy, G. J., Hochedlinger, K., Kotton, D. N., & Mostoslavsky, G. (2009) · Stem Cells · doi:10.1634/stemcells.2008-1075 · Cited by 676
Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells
Mavilio, F., Pellegrini, G., Ferrari, S., Nunzio, F. D., Iorio, E. D., Recchia, A., Maruggi, G., Ferrari, G., Provasi, E., Bonini, C., et al. (2006) · Nature Medicine · doi:10.1038/nm1504 · Cited by 668
Generation of transgene-free lung disease-specific human induced pluripotent stem cells using a single excisable lentiviral stem cell cassette
Somers, A., Jean, J.-C., Sommer, C. A., Omari, A., Ford, C. C., Mills, J. A., Ying, L., Sommer, A. G., Jean, J. M., Smith, B. W., et al. (2010) · Stem Cells · doi:10.1002/stem.495 · Cited by 423
iPS cells: a game changer for future medicine
Inoue, H., Nagata, N., Kurokawa, H., & Yamanaka, S. (2014) · The EMBO Journal · doi:10.1002/embj.201387098 · Cited by 414
In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases
Sebastiano, V., Maeder, M. L., Angstman, J. F., Haddad, B., Khayter, C., Yeo, D. T., Goodwin, M. J., Hawkins, J. S., Ramirez, C. L., Batista, L. F. Z., et al. (2011) · Stem Cells · doi:10.1002/stem.718 · Cited by 311
Self-citation

Show all 39 references

Research works printed but not linked: 1

Siprashvili Z., Nguyen N., Gorell E., Khuu P., Furukawa L., Lorenz H., Leung T., Keene D., Khavari P., Marinkovich M., Lane A., Phase I clinical trial of genetically corrected autologous epidermal keratinocytes for recessive dystrophic epidermolysis bullosa. J. Invest. Dermatol. 134, S75 (2014).

Sorted by citations

Wound repair and regeneration: Mechanisms, signaling, and translation
Eming, S. A., Martin, P., & Tomic-Canic, M. (2014) · Science Translational Medicine · doi:10.1126/scitranslmed.3009337 · Cited by 3,562
Genome-editing Technologies for Gene and Cell Therapy
Maeder, M. L., & Gersbach, C. A. (2016) · Molecular Therapy · doi:10.1038/mt.2016.10 · Cited by 736
Advanced drug delivery systems and artificial skin grafts for skin wound healing
Kim, H. S., Sun, X., Lee, J., Kim, H., Fu, X., & Leong, K. W. (2018) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2018.12.014 · Cited by 639
ApoE2, ApoE3, and ApoE4 Differentially Stimulate APP Transcription and Aβ Secretion
Huang, Y.-W. A., Zhou, B., Wernig, M., & Südhof, T. C. (2017) · Cell · doi:10.1016/j.cell.2016.12.044 · Cited by 528
Self-citation
Induced Pluripotent Stem Cells Meet Genome Editing
Hockemeyer, D., & Jaenisch, R. (2016) · Cell Stem Cell · doi:10.1016/j.stem.2016.04.013 · Cited by 499
“, 2016) and to derive isogeneic cell pairs of COL7A1-corrected iPSCs derived from patients with dystrophic epidermolysis bullosa (Sebastiano et al., 2014).”
Background
Comprehensive comparison of Pacific Biosciences and Oxford Nanopore Technologies and their applications to transcriptome analysis
Weirather, J. L., de Cesare, M., Wang, Y., Piazza, P., Sebastiano, V., Wang, X.-J., Buck, D., & Au, K. F. (2017) · F1000Research · doi:10.12688/f1000research.10571.2 · Cited by 418
Self-citation
Generation of pure GABAergic neurons by transcription factor programming
Yang, N., Chanda, S., Marro, S., Ng, Y.-H., Janas, J. A., Haag, D., Ang, C. E., Tang, Y., Flores, Q., Mall, M., et al. (2017) · Nature Methods · doi:10.1038/nmeth.4291 · Cited by 341
Self-citation
Tissue Stem Cells: Architects of Their Niches
Fuchs, E., & Blau, H. M. (2020) · Cell Stem Cell · doi:10.1016/j.stem.2020.09.011 · Cited by 332
Stem Cells in Wound Healing: The Future of Regenerative Medicine? A Mini-Review
Duscher, D., Barrera, J. A., Wong, V., Maan, Z. N., Whittam, A. J., Januszyk, M., & Gurtner, G. C. (2015) · Gerontology · doi:10.1159/000381877 · Cited by 326
Stem Cells in Skin Regeneration, Wound Healing, and Their Clinical Applications
Ojeh, N. O., Pastar, I., Tomic‐Canic, M., & Stojadinović, O. (2015) · International Journal of Molecular Sciences · doi:10.3390/ijms161025476 · Cited by 310
Current status of pluripotent stem cells: moving the first therapies to the clinic
Kimbrel, E. A., & Lanza, R. (2015) · Nature Reviews Drug Discovery · doi:10.1038/nrd4738 · Cited by 275
Stem Cells in the Treatment of Disease
Blau, H. M., & Daley, G. Q. (2019) · New England Journal of Medicine · doi:10.1056/nejmra1716145 · Cited by 235
Advances of Stem Cell Therapeutics in Cutaneous Wound Healing and Regeneration
Kanji, S., & Das, H. (2017) · Mediators of Inflammation · doi:10.1155/2017/5217967 · Cited by 223
Anisotropic Rod‐Shaped Particles Influence Injectable Granular Hydrogel Properties and Cell Invasion
Qazi, T. H., Wu, J., Muir, V. G., Weintraub, S., Gullbrand, S. E., Lee, D., Issadore, D., & Burdick, J. A. (2021) · Advanced Materials · doi:10.1002/adma.202109194 · Cited by 181
Stem cell therapies for wound healing
Kosaric, N., Kiwanuka, H., & Gurtner, G. C. (2019) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2019.1596257 · Cited by 173
Differential Signaling Mediated by ApoE2, ApoE3, and ApoE4 in Human Neurons Parallels Alzheimer's Disease Risk
Huang, Y.-W. A., Zhou, B., Nabet, A. M., Wernig, M., & Südhof, T. C. (2019) · The Journal of Neuroscience · doi:10.1523/jneurosci.2994-18.2019 · Cited by 152
Self-citation
Stem Cells and Engineered Scaffolds for Regenerative Wound Healing
Dash, B. C., Xu, Z., Lin, L. H., Koo, A. B., Ndon, S. U., Francois Berthiaume, B., Dardik, A., & Hsia, H. C. (2018) · Bioengineering · doi:10.3390/bioengineering5010023 · Cited by 141
Generation and characterization of hair-bearing skin organoids from human pluripotent stem cells
Lee, J., van der Valk, W. H., Serdy, S. A., Deakin, C., Kim, J., Le, A. P., & Koehler, K. R. (2022) · Nature Protocols · doi:10.1038/s41596-022-00681-y · Cited by 132
Mesenchymal stem cells: potential for therapy and treatment of chronic non-healing skin wounds
Marfia, G., Navone, S. E., Vito, C. D., Ughi, N., Tabano, S. M., Miozzo, M. R., Tremolada, C., Bolla, G. B., Crotti, C., Ingegnoli, F., et al. (2015) · Organogenesis · doi:10.1080/15476278.2015.1126018 · Cited by 128
Basement membrane collagens and disease mechanisms
Gatseva, A., Sin, Y. Y., Brezzo, G., & Agtmael, T. V. (2019) · Essays in Biochemistry · doi:10.1042/ebc20180071 · Cited by 126
Induced pluripotent stem cells from human revertant keratinocytes for the treatment of epidermolysis bullosa
Umegaki-Arao, N., Pasmooij, A. M. G., Itoh, M., Cerise, J. E., Guo, Z., Levy, B., Gostyński, A., Rothman, L. R., Jonkman, M. F., & Christiano, A. M. (2014) · Science Translational Medicine · doi:10.1126/scitranslmed.3009342 · Cited by 123
Delivery systems of current biologicals for the treatment of chronic cutaneous wounds and severe burns
Xue, M., Zhao, R., Lin, H., & Jackson, C. (2018) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2018.03.002 · Cited by 124
Autologous Induced Pluripotent Stem Cell–Based Cell Therapies: Promise, Progress, and Challenges
Madrid, M., Sumen, C., Aivio, S., & Saklayen, N. (2021) · Current Protocols · doi:10.1002/cpz1.88 · Cited by 121
CRISPR/Cas9-based targeted genome editing for correction of recessive dystrophic epidermolysis bullosa using iPS cells
Jackow, J., Guo, Z., Hansen, C. S., Abaci, H. E., Doucet, Y., Shin, J. U., Hayashi, R., DeLorenzo, D., Kabata, Y., Shinkuma, S., et al. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1907081116 · Cited by 125
Genome Editing: A New Approach to Human Therapeutics
Porteus, M. H. (2015) · The Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev-pharmtox-010814-124454 · Cited by 118
Human genetic variation alters CRISPR-Cas9 on- and off-targeting specificity at therapeutically implicated loci
Lessard, S., Francioli, L. C., Alföldi, J., Tardif, J., Ellinor, P. T., MacArthur, D. G., Lettre, G., Orkin, S. H., & Canver, M. C. (2017) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1714640114 · Cited by 115
Phase 1/2a clinical trial of gene-corrected autologous cell therapy for recessive dystrophic epidermolysis bullosa
Eichstadt, S. L., Barriga, M., Ponakala, A., Teng, C., Nguyen, N. T., Siprashvili, Z., Nazaroff, J., Gorell, E. S., Chiou, A. S., Taylor, L. S., et al. (2019) · JCI Insight · doi:10.1172/jci.insight.130554 · Cited by 112
Self-citation
The Efficacy of Stem Cells in Wound Healing: A Systematic Review
Farabi, B., Roster, K., Hirani, R., Tepper, K., Atak, M. F., & Safai, B. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25053006 · Cited by 119
The Future of Regenerative Medicine: Cell Therapy Using Pluripotent Stem Cells and Acellular Therapies Based on Extracellular Vesicles
Jarrige, M., Frank, E., Herardot, E., Martineau, S., Darle, A., Benabidès, M., Domingues, S., Chose, O., Habeler, W., Lorant, J., et al. (2021) · Cells · doi:10.3390/cells10020240 · Cited by 107
Next generation human skin constructs as advanced tools for drug development
Abaci, H., Guo, Z., Doucet, Y., Jacków, J., & Christiano, A. (2017) · Experimental Biology and Medicine · doi:10.1177/1535370217712690 · Cited by 104
High-Throughput In Vitro , Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction
Westhaus, A., Cabanes-Creus, M., Rybicki, A., Baltazar, G., Navarro, R. G., Zhu, E., Drouyer, M., Knight, M., Albu, R. F., Ng, B. H., et al. (2020) · Human Gene Therapy · doi:10.1089/hum.2019.264 · Cited by 102
Self-citation
COL7A1 Editing via CRISPR/Cas9 in Recessive Dystrophic Epidermolysis Bullosa
Hainzl, S., Peking, P., Köcher, T., Murauer, E. M., Larcher, F., Río, M. D., Duarte, B., Steiner, M. J., Klausegger, A., Bauer, J. W., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.07.005 · Cited by 103
Efficient in vivo gene editing using ribonucleoproteins in skin stem cells of recessive dystrophic epidermolysis bullosa mouse model
Wu, W., Lü, Z., Li, F., Wang, W., Qian, N., Duan, J., Zhang, Y., Wang, F., & Chen, T. (2017) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1614775114 · Cited by 103
Pathomechanisms of Altered Wound Healing in Recessive Dystrophic Epidermolysis Bullosa
Cianfarani, F., Zambruno, G., Castiglia, D., & Odorisio, T. (2017) · American Journal Of Pathology · doi:10.1016/j.ajpath.2017.03.003 · Cited by 98
Epidermolysis bullosa: Advances in research and treatment
Prodinger, C. M., Reichelt, J., Bauer, J. W., & Laimer, M. (2019) · Experimental Dermatology · doi:10.1111/exd.13979 · Cited by 105
Base Editor Correction of COL7A1 in Recessive Dystrophic Epidermolysis Bullosa Patient-Derived Fibroblasts and iPSCs
Osborn, M. J., Newby, G. A., McElroy, A. N., Knipping, F., Nielsen, S. C., Riddle, M. J., Xia, L., Chen, W., Eide, C. R., Webber, B. R., et al. (2019) · Journal of Investigative Dermatology · doi:10.1016/j.jid.2019.07.701 · Cited by 100
CRISPR/Cas9-based genetic correction for recessive dystrophic epidermolysis bullosa
Webber, B. R., Osborn, M. J., McElroy, A. N., Twaroski, K., Lonetree, C.-L., DeFeo, A. P., Xia, L., Eide, C. R., Lees, C. J., McElmurry, R. T., et al. (2016) · npj Regenerative Medicine · doi:10.1038/npjregenmed.2016.14 · Cited by 98
TFAP2C- and p63-Dependent Networks Sequentially Rearrange Chromatin Landscapes to Drive Human Epidermal Lineage Commitment
Li, L., Wang, Y., Torkelson, J. L., Shankar, G., Pattison, J. M., Zhen, H. H., Fang, F., Duren, Z., Xin, J., Gaddam, S., et al. (2019) · Cell stem cell · doi:10.1016/j.stem.2018.12.012 · Cited by 92
Self-citation
Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa
Lwin, S. M., Syed, F., Di, W., Kadiyirire, T., Liu, L., Guy, A., Petrova, A., Abdul‐Wahab, A., Reid, F., Phillips, R., et al. (2019) · JCI Insight · doi:10.1172/jci.insight.126243 · Cited by 93
Skin organoids: a new human model for developmental and translational research
Lee, J., & Koehler, K. R. (2021) · Experimental Dermatology · doi:10.1111/exd.14292 · Cited by 85
Gene Therapy for Epidermolysis Bullosa
Marinkovich, M. P., & Tang, J. Y. (2019) · Journal of Investigative Dermatology · doi:10.1016/j.jid.2018.11.036 · Cited by 88
Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa
Bremer, J., Bornert, O., Nyström, A., Gostyński, A., Jonkman, M. F., Aartsma-Rus, A. M., van den Akker, P. C., & Pasmooij, A. M. G. (2016) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2016.87 · Cited by 79
Progress toward Treatment and Cure of Epidermolysis Bullosa: Summary of the DEBRA International Research Symposium EB2015
Uitto, J., Bruckner‐Tuderman, L., Christiano, A. M., McGrath, J. A., Has, C., South, A. P., Kopelan, B., & Robinson, E. C. (2016) · Journal of Investigative Dermatology · doi:10.1016/j.jid.2015.10.050 · Cited by 76
Stem cells and growth factors-based delivery approaches for chronic wound repair and regeneration: A promise to heal from within
Sharma, P. M., Kumar, A., Dey, A. D., Behl, T., & Chadha, S. (2021) · Life Sciences · doi:10.1016/j.lfs.2020.118932 · Cited by 70
Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair
Izmiryan, A., Ganier, C., Bovolenta, M., Schmitt, A., Mavilio, F., & Hovnanian, A. (2018) · Molecular Therapy Nucleic Acids · doi:10.1016/j.omtn.2018.06.008 · Cited by 68
EB2017—Progress in Epidermolysis Bullosa Research toward Treatment and Cure
Uitto, J., Bruckner‐Tuderman, L., McGrath, J. A., Riedl, R., & Robinson, C. (2018) · Journal of Investigative Dermatology · doi:10.1016/j.jid.2017.12.016 · Cited by 71
Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic Progenitors
Kim, J., Magli, A., Chan, S., Oliveira, V. K. P., Wu, J.-B., Darabi, R., Kyba, M., & Perlingeiro, R. (2017) · Stem Cell Reports · doi:10.1016/j.stemcr.2017.04.022 · Cited by 57
Genetically corrected iPSCs as cell therapy for recessive dystrophic epidermolysis bullosa
Wenzel, D., Bayerl, J., Nyström, A., Bruckner-Tuderman, L., Meixner, A., & Penninger, J. M. (2014) · Science Translational Medicine · doi:10.1126/scitranslmed.3010083 · Cited by 62
Considerations for the development of iPSC-derived cell therapies: a review of key challenges by the JSRM-ISCT iPSC Committee
Madrid, M., Lakshmipathy, U., Zhang, X., Bharti, K., Wall, D. M., Sato, Y., Muschler, G. F., Ting, A. E., Smith, N., Deguchi, S., et al. (2024) · Cytotherapy · doi:10.1016/j.jcyt.2024.05.022 · Cited by 67
Stem cells, niches and scaffolds: Applications to burns and wound care
Watt, S. M., & Pleat, J. (2017) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2017.10.012 · Cited by 63
Retinoic acid and BMP4 cooperate with p63 to alter chromatin dynamics during surface epithelial commitment
Pattison, J. M., Melo, S. P., Piekos, S. N., Torkelson, J. L., Bashkirova, E., Mumbach, M. R., Rajasingh, C. M., Zhen, H. H., Li, L., Liaw, E. J., et al. (2018) · Nature Genetics · doi:10.1038/s41588-018-0263-0 · Cited by 62
Self-citation
Investigational Treatments for Epidermolysis Bullosa
Hou, P., Wang, H., Abhee, S. S., Tu, W., McGrath, J. A., & Hsu, C. (2021) · American Journal of Clinical Dermatology · doi:10.1007/s40257-021-00626-3 · Cited by 67
Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes
Chamorro, C., Mencía, Á., Almarza, D., Duarte, B., Büning, H., Sallach, J., Haußer, I., Río, M. D., Larcher, F., & Murillas, R. (2016) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2016.19 · Cited by 61
The PfAP2‐G2 transcription factor is a critical regulator of gametocyte maturation
Singh, S., Santos, J., Orchard, L., Yamada, N., van Biljon, R., Painter, H. J., Mahony, S., & Llinás, M. (2020) · Molecular Microbiology · doi:10.1111/mmi.14676 · Cited by 61
Patient-Specific iPSC-Derived Endothelial Cells Provide Long-Term Phenotypic Correction of Hemophilia A
Olgasi, C., Talmon, M., Merlin, S., Cucci, A., Richaud‐Patín, Y., Ranaldo, G., Colangelo, D., Scipio, F. D., Berta, G. N., Borsotti, C., et al. (2018) · Stem Cell Reports · doi:10.1016/j.stemcr.2018.10.012 · Cited by 58
CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells
Kim, E. J., Kang, K. H., & Ju, J. H. (2017) · The Korean Journal of Internal Medicine · doi:10.3904/kjim.2016.198 · Cited by 58
Extracellular vesicles modulate key signalling pathways in refractory wound healing
Yang, B., Lin, Y., Huang, Y., Zhu, N., & Shen, Y. (2023) · Burns & Trauma · doi:10.1093/burnst/tkad039 · Cited by 60
Efficient Gene Reframing Therapy for Recessive Dystrophic Epidermolysis Bullosa with CRISPR/Cas9
Takashima, S., Shinkuma, S., Fujita, Y., Nomura, T., Ujiie, H., Natsuga, K., Iwata, H., Nakamura, H., Vorobyev, A., Abe, R., et al. (2019) · Journal of Investigative Dermatology · doi:10.1016/j.jid.2019.02.015 · Cited by 57
DNA repair fidelity in stem cell maintenance, health, and disease
Mani, C., Reddy, P. H., & Palle, K. (2019) · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease · doi:10.1016/j.bbadis.2019.03.017 · Cited by 56
Advancement in management of epidermolysis bullosa
Cohn, H. I., & Teng, J. M. C. (2016) · Current Opinion in Pediatrics · doi:10.1097/mop.0000000000000380 · Cited by 58
Gene-Corrected Fibroblast Therapy for Recessive Dystrophic Epidermolysis Bullosa using a Self-Inactivating COL7A1 Retroviral Vector
Jackow, J., Titeux, M., Portier, S., Charbonnier, S., Gânier, C., Gaucher, S., & Hovnanian, A. (2016) · Journal of Investigative Dermatology · doi:10.1016/j.jid.2016.02.811 · Cited by 54
Lentiviral Engineered Fibroblasts Expressing Codon-Optimized COL7A1 Restore Anchoring Fibrils in RDEB
Georgiadis, C., Syed, F., Petrova, A., Abdul‐Wahab, A., Lwin, S. M., Farzaneh, F., Chan, L., Ghani, S., Fleck, R. A., Glover, L., et al. (2015) · Journal of Investigative Dermatology · doi:10.1038/jid.2015.364 · Cited by 54
Cell therapy for basement membrane-linked diseases
Nyström, A., Bornert, O., & Kühl, T. (2016) · Matrix Biology · doi:10.1016/j.matbio.2016.07.012 · Cited by 50
Assessment of the Timing of Milestone Clinical Events in Patients With Epidermolysis Bullosa From North America
Feinstein, J. A., Jambal, P., Peoples, K., Lucky, A. W., Khuu, P. T., Tang, J. Y., Lara‐Corrales, I., Pope, E., Wiss, K., Hook, K. P., et al. (2018) · JAMA Dermatology · doi:10.1001/jamadermatol.2018.4673 · Cited by 49
Keratinocytes Derived from Patient-Specific Induced Pluripotent Stem Cells Recapitulate the Genetic Signature of Psoriasis Disease
Ali, G., Elsayed, A. K., Nandakumar, M., Bashir, M., Younis, I., Aqel, Y. W. A., Memon, B., Temanni, R., Abubaker, F., Taheri, S., et al. (2020) · Stem Cells and Development · doi:10.1089/scd.2019.0150 · Cited by 47
Pluripotent Stem Cells: The Last 10 Years
Kimbrel, E. A., & Lanza, R. (2016) · Regenerative Medicine · doi:10.2217/rme-2016-0117 · Cited by 45
Mosaicism in Cutaneous Disorders
Lim, Y. H., Moscato, Z. M., & Choate, K. A. (2017) · Annual Review of Genetics · doi:10.1146/annurev-genet-121415-121955 · Cited by 45
Students’ Perspectives on Interprofessional Teamwork Before and After an Interprofessional Pain Education Course
Simko, L. C., Rhodes, D. C., McGinnis, K. A., & Fiedor, J. (2017) · American Journal of Pharmaceutical Education · doi:10.5688/ajpe816104 · Cited by 42
Biomaterial-mediated reprogramming of monocytes via microparticle phagocytosis for sustained modulation of macrophage phenotype
Wofford, K. L., Singh, B. S., Cullen, D. K., & Spiller, K. L. (2020) · Acta Biomaterialia · doi:10.1016/j.actbio.2019.11.021 · Cited by 39
Gut bacteria Akkermansia elicit a specific IgG response in CSF of patients with MS
Vallino, A., Santos, A. D., Mathé, C., Garcia, A., Morille, J., Dugast, E., Shah, S. P., Héry-Arnaud, G., Guilloux, C.-A., Gleeson, P. J., et al. (2020) · Neurology Neuroimmunology & Neuroinflammation · doi:10.1212/nxi.0000000000000688 · Cited by 41
Large-scale production of lentiviral vector in a closed system hollow fiber bioreactor
Sheu, J. I., Beltzer, J., Fury, B., Wilczek, K., Tobin, S., Falconer, D., Nolta, J. A., & Bauer, G. (2015) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2015.20 · Cited by 39
Self-citation
Combining Induced Pluripotent Stem Cells and Genome Editing Technologies for Clinical Applications
Chang, C.-Y., Ting, H.-C., Su, H., & Jeng, J. (2018) · Cell Transplantation · doi:10.1177/0963689718754560 · Cited by 39
Engineered Epidermal Progenitor Cells Can Correct Diet-Induced Obesity and Diabetes
Yue, J., Gou, X., Li, Y., Wicksteed, B. L., & Wu, X. (2017) · Cell stem cell · doi:10.1016/j.stem.2017.06.016 · Cited by 37
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality
Cabanes‐Creus, M., Ginn, S. L., Amaya, A. K., Liao, S. H. Y., Westhaus, A., Hallwirth, C. V., Wilmott, P., Ward, J., Dilworth, K. L., Santilli, G., et al. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.10.016 · Cited by 36
Self-citation
Toward Combined Cell and Gene Therapy for GenodermatosesPreprint
Rosa, L. D., Latella, M. C., Seconetti, A. S., Cattelani, C., Bauer, J. W., Bondanza, S., & Luca, M. D. (2019) · Cold Spring Harbor Perspectives in Biology · doi:10.1101/cshperspect.a035667 · Cited by 37
Genetically modified cell sheets in regenerative medicine and tissue engineering
Jiang, Z., Li, N., Zhu, D., Ren, L., Shao, Q., Ke, Y., & Yang, G. (2021) · Biomaterials · doi:10.1016/j.biomaterials.2021.120908 · Cited by 37
Context-Dependent Strategies for Enhanced Genome Editing of Genodermatoses
March, O. P., Köcher, T., & Koller, U. (2020) · Cells · doi:10.3390/cells9010112 · Cited by 36
Activities of daily living with bionic arm improved by combination training and latching filter in prosthesis control comparison
Paskett, M. D., Brinton, M. R., Hansen, T. C., George, J. A., Davis, T. S., Duncan, C. C., & Clark, G. A. (2021) · Journal of NeuroEngineering and Rehabilitation · doi:10.1186/s12984-021-00839-x · Cited by 36
Current and Future Perspectives of Stem Cell Therapy in Dermatology
Prodinger, C. M., Reichelt, J., Bauer, J. W., & Laimer, M. (2017) · Annals of Dermatology · doi:10.5021/ad.2017.29.6.667 · Cited by 37
dsRNA-Seq: Identification of Viral Infection by Purifying and Sequencing dsRNA
Decker, C. J., Steiner, H. R., Hoon‐Hanks, L. L., Morrison, J. H., Haist, K. C., Stabell, A. C., Poeschla, E. M., Morrison, T. E., Stenglein, M. D., Sawyer, S. L., et al. (2019) · Viruses · doi:10.3390/v11100943 · Cited by 33
Correction of Recessive Dystrophic Epidermolysis Bullosa by Transposon-Mediated Integration of COL7A1 in Transplantable Patient-Derived Primary Keratinocytes
Latella, M. C., Cocchiarella, F., Rosa, L. D., Turchiano, G., Gonçalves, M. A. F. V., Larcher, F., Luca, M. D., & Recchia, A. (2016) · Journal of Investigative Dermatology · doi:10.1016/j.jid.2016.11.038 · Cited by 33
Engineering the next generation of allogeneic CAR cells: iPSCs as a scalable and editable platform
Fang, Y., Chen, Y., & Li, Y.-R. (2025) · Stem Cell Reports · doi:10.1016/j.stemcr.2025.102515 · Cited by 36
Bioactive functional scaffolds for stem cells delivery in wound healing and skin regeneration
Hazrati, R., Davaran, S., & Omidi, Y. (2022) · Reactive and Functional Polymers · doi:10.1016/j.reactfunctpolym.2022.105233 · Cited by 33
Bone marrow adipocytes drive the development of tissue invasive Ly6Chigh monocytes during obesity
Boroumand, P., Prescott, D., Mukherjee, T., Bilan, P. J., Wong, M. D., Shen, J., Tattoli, I., Zhou, Y., Li, A., Sivasubramaniyam, T., et al. (2022) · eLife · doi:10.7554/elife.65553 · Cited by 35
Gibbin mesodermal regulation patterns epithelial development
Collier, A., Liu, A., Torkelson, J., Pattison, J. M., Gaddam, S., Zhen, H., Patel, T., McCarthy, K., Ghanim, H. Y., & Oro, A. (2022) · Nature · doi:10.1038/s41586-022-04727-9 · Cited by 27
Self-citation
Meganuclease-Mediated COL7A1 Gene Correction for Recessive Dystrophic Epidermolysis Bullosa
Izmiryan, A., Danos, O., & Hovnanian, A. (2016) · Journal of Investigative Dermatology · doi:10.1016/j.jid.2015.11.028 · Cited by 32
Elexacaftor/VX-445–mediated CFTR interactome remodeling reveals differential correction driven by mutation-specific translational dynamics
Kim, M., McDonald, E. F., Sabusap, C. M. P., Timalsina, B., Joshi, D., Hong, J. S., Rab, A., Sorscher, E. J., & Plate, L. (2023) · Journal of Biological Chemistry · doi:10.1016/j.jbc.2023.105242 · Cited by 28
A mouse model of Weaver syndrome displays overgrowth and excess osteogenesis reversible with KDM6A/6B inhibition
Gao, C. W., Lin, W., Riddle, R. C., Kushwaha, P., Boukas, L., Bjornsson, H. T., Hansen, K. D., & Fahrner, J. A. (2023) · JCI Insight · doi:10.1172/jci.insight.173392 · Cited by 33
Engineering edgeless human skin with enhanced biomechanical properties
Pappalardo, A., Cespedes, D. A., Fang, S., Herschman, A. R., Jeon, E. Y., Myers, K. M., Kysar, J. W., & Abaci, H. E. (2023) · Science Advances · doi:10.1126/sciadv.ade2514 · Cited by 30
Therapies for genetic extracellular matrix diseases of the skin
Nyström, A., Bernasconi, R., & Bornert, O. (2017) · Matrix Biology · doi:10.1016/j.matbio.2017.12.010 · Cited by 29
Footprint-free gene mutation correction in induced pluripotent stem cell (iPSC) derived from recessive dystrophic epidermolysis bullosa (RDEB) using the CRISPR/Cas9 and piggyBac transposon system
Itoh, M., Kawagoe, S., Tamai, K., Nakagawa, H., Asahina, A., & Okano, H. J. (2020) · Journal of Dermatological Science · doi:10.1016/j.jdermsci.2020.04.004 · Cited by 30
The potential of gene therapy for recessive dystrophic epidermolysis bullosa*
Subramaniam, K. S., Antoniou, M. N., McGrath, J. A., & Lwin, S. M. (2021) · British Journal of Dermatology · doi:10.1111/bjd.20910 · Cited by 28
Direct somatic lineage conversion
Tanabe, K., Haag, D., & Wernig, M. (2015) · Philosophical Transactions of the Royal Society B Biological Sciences · doi:10.1098/rstb.2014.0368 · Cited by 29
Self-citation
Revertant mosaicism in genodermatoses
Lim, Y. H., Fisher, J. M., & Choate, K. A. (2017) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-017-2468-2 · Cited by 28
A non-viral and selection-free COL7A1 HDR approach with improved safety profile for dystrophic epidermolysis bullosa
Köcher, T., Bischof, J., Haas, S. A., March, O. P., Liemberger, B., Hainzl, S., Illmer, J., Hoog, A., Muigg, K., Binder, H., et al. (2021) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2021.05.015 · Cited by 27
Clinical Perspectives of Gene-Targeted Therapies for Epidermolysis Bullosa
Welponer, T., Prodinger, C. M., Hofbauer, J. P., Hintersteininger, A., Breitenbach‐Koller, H., Bauer, J. W., & Laimer, M. (2021) · Dermatology and Therapy · doi:10.1007/s13555-021-00561-5 · Cited by 29
Genetic Disorders of the Extracellular Matrix: From Cell and Gene Therapy to Future Applications in Regenerative Medicine
Chakravarti, S., Enzo, E., de Barros, M. R. M., Maffezzoni, M. B. R., & Pellegrini, G. (2022) · Annual Review of Genomics and Human Genetics · doi:10.1146/annurev-genom-083117-021702 · Cited by 28
Allele-specific silencing of EEC p63 mutant R304W restores p63 transcriptional activity
Novelli, F., Lena, A. M., Panatta, E., Nasser, W., Shalom‐Feuerstein, R., Candi, E., & Melino, G. (2016) · Cell Death and Disease · doi:10.1038/cddis.2016.118 · Cited by 26
Inside out: regenerative medicine for recessive dystrophic epidermolysis bullosa
Oever, M. V., Twaroski, K., Osborn, M. J., Wagner, J. E., & Tolar, J. (2017) · Pediatric Research · doi:10.1038/pr.2017.244 · Cited by 28
Generation of Keratinocytes from Human Induced Pluripotent Stem Cells Under Defined Culture Conditions
Sah, S. K., Kanaujiya, J. K., Chen, I., & Reichenberger, E. (2020) · Cellular Reprogramming · doi:10.1089/cell.2020.0046 · Cited by 28

Authors: Sebastiano, V., Zhen, H. H., Haddad, B., Bashkirova, E., Melo, S. P., Wang, P., Leung, T. L., Siprashvili, Z., Tichy, A., Li, J., Ameen, M., Hawkins, J., Lee, S., Li, L., Schwertschkow, A., Bauer, G., Lisowski, L., Kay, M. A., Kim, S. K., Lane, A. T., Wernig, M., & Oro, A. E.
Affiliations: Institute for Stem Cell Biology and Regenerative Medicine (US)
Stanford Medicine (US)
Stanford University (US)
University of California, Davis (US)
Corresponding author: Sebastiano, Vittorio
Zhen, Hanson Hui
Wernig, Marius
Oro, Anthony E.
ORCID: Sebastiano, Vittorio
Wang, Pei
Leung, Thomas L.
Siprashvili, Zurab
Li, Jiang
Li, Lingjie
Lisowski, Leszek
Kay, Mark A.
Kim, Seung K.
Wernig, Marius
Oro, Anthony E.
Venue: Science Translational Medicine
Publisher: American Association for the Advancement of Science (AAAS)
Published: 2014-11-26
Volume: 6
Issue: 264
Pages: 264ra163-264ra163
ISSN: 1946-6234 (ISSN-L)
1946-6242
DOI: 10.1126/scitranslmed.3009540
PMC: PMC4428910
OpenAlex: W2085600205
Cited by: 213
References: 39
Citations by year: 2026: 2 · 2025: 10 · 2024: 9 · 2023: 12 · 2022: 17 · 2021: 29 · 2020: 15 · 2019: 21 · 2018: 23 · 2017: 27 · 2016: 22 · 2015: 23 · 2014: 3
FWCI: 10.26
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Locations: Science Translational Medicine (publishedVersion)
PubMed (publishedVersion)
Europe PMC (PubMed Central) (acceptedVersion)
PubMed Central (acceptedVersion)
Funders: Howard Hughes Medical Institute
National Heart, Lung, and Blood Institute (R01HL064274)
National Institute of Arthritis and Musculoskeletal and Skin Diseases (R01AR055914, K08AR066661, T32AR007422)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Pluripotent Stem Cells Research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Cell Biology › Skin and Cellular Biology Research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
Keywords: recessive dystrophic epidermolysis bullosa, COL7A1, type VII collagen, induced pluripotent stem cells, genome editing, keratinocytes, organotypic culture, genodermatosis, squamous cell carcinoma
MeSH terms: Animals, Base Sequence, Genes, Recessive, Humans, Molecular Sequence Data, Mutation, Genetic Therapy, Keratinocytes, Genome, Human, Epidermolysis Bullosa Dystrophica, Sequence Analysis, DNA, Genetic Predisposition to Disease, Collagen Type VII, Mice, Induced Pluripotent Stem Cells, Homologous Recombination

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review

Human COL7A1 -corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa

Abstract

Overview

Integrity signals

Verification

Claims the paper makes

Claims other papers make about this one

Self-check

Citations

Cites

Cited by

Related

Metadata

Record

About these checks