Toward Precision Medicine with Human Pluripotent Stem Cells for Diabetes
<div><p>Although genome profiling provides important genetic and phenotypic details for applying precision medicine to diabetes, it is imperative to integrate in vitro human cell models, accurately recapitulating the genetic alterations associated with diabetes. The absence of the approp...
محفوظ في:
| المؤلف الرئيسي: | |
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| مؤلفون آخرون: | |
| منشور في: |
2022
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| الموضوعات: | |
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| _version_ | 1864513519258435584 |
|---|---|
| author | Bushra Memon (4792767) |
| author2 | Essam M Abdelalim (18300838) |
| author2_role | author |
| author_facet | Bushra Memon (4792767) Essam M Abdelalim (18300838) |
| author_role | author |
| dc.creator.none.fl_str_mv | Bushra Memon (4792767) Essam M Abdelalim (18300838) |
| dc.date.none.fl_str_mv | 2022-05-28T03:00:00Z |
| dc.identifier.none.fl_str_mv | 10.1093/stcltm/szac030 |
| dc.relation.none.fl_str_mv | https://figshare.com/articles/journal_contribution/Toward_Precision_Medicine_with_Human_Pluripotent_Stem_Cells_for_Diabetes/25539622 |
| dc.rights.none.fl_str_mv | CC BY 4.0 info:eu-repo/semantics/openAccess |
| dc.subject.none.fl_str_mv | Biological sciences Biochemistry and cell biology Plant biology hPSCs insulin-secreting cells pathogenesis drug development personalized therapy |
| dc.title.none.fl_str_mv | Toward Precision Medicine with Human Pluripotent Stem Cells for Diabetes |
| dc.type.none.fl_str_mv | Text Journal contribution info:eu-repo/semantics/publishedVersion text contribution to journal |
| description | <div><p>Although genome profiling provides important genetic and phenotypic details for applying precision medicine to diabetes, it is imperative to integrate in vitro human cell models, accurately recapitulating the genetic alterations associated with diabetes. The absence of the appropriate preclinical human models and the unavailability of genetically relevant cells substantially limit the progress in developing personalized treatment for diabetes. Human pluripotent stem cells (hPSCs) provide a scalable source for generating diabetes-relevant cells carrying the genetic signatures of the patients. Remarkably, allogenic hPSC-derived pancreatic progenitors and β cells are being used in clinical trials with promising preliminary results. Autologous hiPSC therapy options exist for those with monogenic and type 2 diabetes; however, encapsulation or immunosuppression must be accompanied with in the case of type 1 diabetes. Furthermore, genome-wide association studies-identified candidate variants can be introduced in hPSCs for deciphering the associated molecular defects. The hPSC-based disease models serve as excellent resources for drug development facilitating personalized treatment. Indeed, hPSC-based diabetes models have successfully provided valuable knowledge by modeling different types of diabetes, which are discussed in this review. Herein, we also evaluate their strengths and shortcomings in dissecting the underlying pathogenic molecular mechanisms and discuss strategies for improving hPSC-based disease modeling investigations.</p><p> </p></div><h2>Other Information</h2> <p> Published in: Stem Cells Translational Medicine<br> License: <a href="https://creativecommons.org/licenses/by/4.0/" target="_blank">https://creativecommons.org/licenses/by/4.0/</a><br>See article on publisher's website: <a href="https://dx.doi.org/10.1093/stcltm/szac030" target="_blank">https://dx.doi.org/10.1093/stcltm/szac030</a></p> |
| eu_rights_str_mv | openAccess |
| id | Manara2_a409812474e28e802c35700b49bbe6d3 |
| identifier_str_mv | 10.1093/stcltm/szac030 |
| network_acronym_str | Manara2 |
| network_name_str | Manara2 |
| oai_identifier_str | oai:figshare.com:article/25539622 |
| publishDate | 2022 |
| repository.mail.fl_str_mv | |
| repository.name.fl_str_mv | |
| repository_id_str | |
| rights_invalid_str_mv | CC BY 4.0 |
| spelling | Toward Precision Medicine with Human Pluripotent Stem Cells for DiabetesBushra Memon (4792767)Essam M Abdelalim (18300838)Biological sciencesBiochemistry and cell biologyPlant biologyhPSCsinsulin-secreting cellspathogenesisdrug developmentpersonalized therapy<div><p>Although genome profiling provides important genetic and phenotypic details for applying precision medicine to diabetes, it is imperative to integrate in vitro human cell models, accurately recapitulating the genetic alterations associated with diabetes. The absence of the appropriate preclinical human models and the unavailability of genetically relevant cells substantially limit the progress in developing personalized treatment for diabetes. Human pluripotent stem cells (hPSCs) provide a scalable source for generating diabetes-relevant cells carrying the genetic signatures of the patients. Remarkably, allogenic hPSC-derived pancreatic progenitors and β cells are being used in clinical trials with promising preliminary results. Autologous hiPSC therapy options exist for those with monogenic and type 2 diabetes; however, encapsulation or immunosuppression must be accompanied with in the case of type 1 diabetes. Furthermore, genome-wide association studies-identified candidate variants can be introduced in hPSCs for deciphering the associated molecular defects. The hPSC-based disease models serve as excellent resources for drug development facilitating personalized treatment. Indeed, hPSC-based diabetes models have successfully provided valuable knowledge by modeling different types of diabetes, which are discussed in this review. Herein, we also evaluate their strengths and shortcomings in dissecting the underlying pathogenic molecular mechanisms and discuss strategies for improving hPSC-based disease modeling investigations.</p><p> </p></div><h2>Other Information</h2> <p> Published in: Stem Cells Translational Medicine<br> License: <a href="https://creativecommons.org/licenses/by/4.0/" target="_blank">https://creativecommons.org/licenses/by/4.0/</a><br>See article on publisher's website: <a href="https://dx.doi.org/10.1093/stcltm/szac030" target="_blank">https://dx.doi.org/10.1093/stcltm/szac030</a></p>2022-05-28T03:00:00ZTextJournal contributioninfo:eu-repo/semantics/publishedVersiontextcontribution to journal10.1093/stcltm/szac030https://figshare.com/articles/journal_contribution/Toward_Precision_Medicine_with_Human_Pluripotent_Stem_Cells_for_Diabetes/25539622CC BY 4.0info:eu-repo/semantics/openAccessoai:figshare.com:article/255396222022-05-28T03:00:00Z |
| spellingShingle | Toward Precision Medicine with Human Pluripotent Stem Cells for Diabetes Bushra Memon (4792767) Biological sciences Biochemistry and cell biology Plant biology hPSCs insulin-secreting cells pathogenesis drug development personalized therapy |
| status_str | publishedVersion |
| title | Toward Precision Medicine with Human Pluripotent Stem Cells for Diabetes |
| title_full | Toward Precision Medicine with Human Pluripotent Stem Cells for Diabetes |
| title_fullStr | Toward Precision Medicine with Human Pluripotent Stem Cells for Diabetes |
| title_full_unstemmed | Toward Precision Medicine with Human Pluripotent Stem Cells for Diabetes |
| title_short | Toward Precision Medicine with Human Pluripotent Stem Cells for Diabetes |
| title_sort | Toward Precision Medicine with Human Pluripotent Stem Cells for Diabetes |
| topic | Biological sciences Biochemistry and cell biology Plant biology hPSCs insulin-secreting cells pathogenesis drug development personalized therapy |