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1.
Cell Stem Cell ; 29(4): 610-619.e5, 2022 04 07.
Artigo em Inglês | MEDLINE | ID: mdl-35395188

RESUMO

Human pluripotent stem cell (hPSC)-derived myogenic progenitor cell (MPC) transplantation is a promising therapeutic approach for a variety of degenerative muscle disorders. Here, using an MPC-specific fluorescent reporter system (PAX7::GFP), we demonstrate that hPSC-derived MPCs can contribute to the regeneration of myofibers in mice following local injury and in mice deficient of dystrophin (mdx). We also demonstrate that a subset of PAX7::GFP MPCs engraft within the basal lamina of regenerated myofibers, adopt a quiescent state, and contribute to regeneration upon reinjury and in mdx mouse models. This subset of PAX7::GFP MPCs undergo a maturation process and remodel their molecular characteristics to resemble those of late-stage fetal MPCs/adult satellite cells following in vivo engraftment. These in-vivo-matured PAX7::GFP MPCs retain a cell-autonomous ability to regenerate and can repopulate in the niche of secondary recipient mice, providing a proof of principle for future hPSC-based cell therapy for muscle disorders.


Assuntos
Células-Tronco Pluripotentes , Células Satélites de Músculo Esquelético , Animais , Diferenciação Celular , Distrofina , Humanos , Camundongos , Camundongos Endogâmicos mdx , Desenvolvimento Muscular , Músculo Esquelético , Mioblastos , Transplante de Células-Tronco
2.
J Patient Cent Res Rev ; 7(1): 8-18, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-32002443

RESUMO

PURPOSE: Shared decision-making (SDM) has a significant role in surgical encounters, where decisions are influenced by both clinician and patient preferences. Herein, we sought to explore surgeons' practices and beliefs about SDM. METHODS: We performed a qualitative study consisting of semi-structured individual interviews with 18 surgeons from private practice and academic surgery practices in Baltimore, Maryland. We purposively sampled participants to maximize diversity of practice type (academic vs private), surgical specialty, gender, and experience level. Interview topics included benefits and challenges to patient involvement in decision-making, communicating uncertainty to patients, and use of decision aids. Interviews were audio-recorded and transcribed. Transcripts were analyzed using content analysis to identify themes. RESULTS: Surgeons were supportive of patients being involved in decision-making, particularly in cases with uncertainty about treatment options. However, surgeons identified SDM as being more appropriate for patients whom surgeons perceived as interested in decision-making involvement and for decisions in which surgeons did not have strong preferences. Additionally, surgeons reported typically presenting only a subset of available options, remaining confident in their ability to filter less suitable options based on intuitive risk assessments. Surgeons differed in their approach to making recommendations, with some guiding patients towards what they saw as the correct or optimal decision while others sought to maintain neutrality and support of the patients' chosen decision. CONCLUSIONS: Many surgeons do not believe SDM is universally optimal for every surgical decision. They instead use assessments of patient disposition or potential clinical uncertainty to guide their perceived appropriateness of using SDM.

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