Literature DB >> 31473142

Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies.

Sridhar Selvaraj1, Michael Kyba2, Rita C R Perlingeiro3.   

Abstract

Pluripotent stem cells (PSCs) represent an attractive cell source for treating muscular dystrophies (MDs) since they easily allow for the generation of large numbers of highly regenerative myogenic progenitors. Using reprogramming technology, patient-specific PSCs have been derived for several types of MDs, and genome editing has allowed correction of mutations, opening the opportunity for their therapeutic application in an autologous transplantation setting. However, there has been limited progress on preclinical studies that validate the therapeutic potential of these gene corrected PSC-derived myogenic progenitors. In this review, we highlight the major research advances, challenges, and future prospects towards the development of PSC-based therapeutics for MDs.
Copyright © 2019 Elsevier Ltd. All rights reserved.

Entities:  

Keywords:  cell therapy; gene correction; muscular dystrophy; myogenic progenitors; pluripotent stem cells

Mesh：

Substances：

Year:  2019        PMID： 31473142      PMCID： PMC6721995          DOI： 10.1016/j.molmed.2019.07.004

Source DB:  PubMed          Journal:  Trends Mol Med        ISSN： 1471-4914            Impact factor:   11.951


  86 in total

1.  Pax7 directs postnatal renewal and propagation of myogenic satellite cells but not their specification.

Authors:  Svetlana Oustanina; Gerd Hause; Thomas Braun
Journal:  EMBO J       Date:  2004-07-29       Impact factor: 11.598

Review 2.  Myoblast transplantation: clinical trials and perspectives. Mini-review.

Authors:  J T Vilquin
Journal:  Acta Myol       Date:  2005-10

3.  Direct isolation of satellite cells for skeletal muscle regeneration.

Authors:  Didier Montarras; Jennifer Morgan; Charlotte Collins; Frédéric Relaix; Stéphane Zaffran; Ana Cumano; Terence Partridge; Margaret Buckingham
Journal:  Science       Date:  2005-09-01       Impact factor: 47.728

4.  Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors.

Authors:  Kazutoshi Takahashi; Shinya Yamanaka
Journal:  Cell       Date:  2006-08-10       Impact factor: 41.582

Review 5.  The dystrophin glycoprotein complex: signaling strength and integrity for the sarcolemma.

Authors:  Karen A Lapidos; Rahul Kakkar; Elizabeth M McNally
Journal:  Circ Res       Date:  2004-04-30       Impact factor: 17.367

6.  Resetting the problem of cell death following muscle-derived cell transplantation: detection, dynamics and mechanisms.

Authors:  Daniel Skuk; Nicolas J Caron; Marlyne Goulet; Brigitte Roy; Jacques P Tremblay
Journal:  J Neuropathol Exp Neurol       Date:  2003-09       Impact factor: 3.685

7.  Derivation of engraftable skeletal myoblasts from human embryonic stem cells.

Authors:  Tiziano Barberi; Michelle Bradbury; Zehra Dincer; Georgia Panagiotakos; Nicholas D Socci; Lorenz Studer
Journal:  Nat Med       Date:  2007-04-08       Impact factor: 53.440

8.  Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs.

Authors:  Maurilio Sampaolesi; Stephane Blot; Giuseppe D'Antona; Nicolas Granger; Rossana Tonlorenzi; Anna Innocenzi; Paolo Mognol; Jean-Lauren Thibaud; Beatriz G Galvez; Ines Barthélémy; Laura Perani; Sara Mantero; Maria Guttinger; Orietta Pansarasa; Chiara Rinaldi; M Gabriella Cusella De Angelis; Yvan Torrente; Claudio Bordignon; Roberto Bottinelli; Giulio Cossu
Journal:  Nature       Date:  2006-11-15       Impact factor: 49.962

9.  Dynamics of myoblast transplantation reveal a discrete minority of precursors with stem cell-like properties as the myogenic source.

Authors:  J R Beauchamp; J E Morgan; C N Pagel; T A Partridge
Journal:  J Cell Biol       Date:  1999-03-22       Impact factor: 10.539

10.  Cell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts.

Authors:  Maurilio Sampaolesi; Yvan Torrente; Anna Innocenzi; Rossana Tonlorenzi; Giuseppe D'Antona; M Antonietta Pellegrino; Rita Barresi; Nereo Bresolin; M Gabriella Cusella De Angelis; Kevin P Campbell; Roberto Bottinelli; Giulio Cossu
Journal:  Science       Date:  2003-07-10       Impact factor: 47.728
View more
  6 in total

1.  Generation of Human iPSC-Derived Myotubes to Investigate RNA-Based Therapies In Vitro.

Authors:  Pablo Herrero-Hernandez; Atze J Bergsma; W W M Pim Pijnappel
Journal:  Methods Mol Biol       Date:  2022

Review 2.  Fukutin-Related Protein: From Pathology to Treatments.

Authors:  Carolina Ortiz-Cordero; Karim Azzag; Rita C R Perlingeiro
Journal:  Trends Cell Biol       Date:  2020-12-01       Impact factor: 20.808

3.  Multilineage Differentiation for Formation of Innervated Skeletal Muscle Fibers from Healthy and Diseased Human Pluripotent Stem Cells.

Authors:  Kilian Mazaleyrat; Cherif Badja; Natacha Broucqsault; Raphaël Chevalier; Camille Laberthonnière; Camille Dion; Lyla Baldasseroni; Claire El-Yazidi; Morgane Thomas; Richard Bachelier; Alexandre Altié; Karine Nguyen; Nicolas Lévy; Jérôme D Robin; Frédérique Magdinier
Journal:  Cells       Date:  2020-06-23       Impact factor: 6.600

4.  Customized bioreactor enables the production of 3D diaphragmatic constructs influencing matrix remodeling and fibroblast overgrowth.

Authors:  Edoardo Maghin; Eugenia Carraro; Daniele Boso; Arben Dedja; Mattia Giagante; Paola Caccin; Raluca Ana-Maria Barna; Silvia Bresolin; Alice Cani; Giulia Borile; Deborah Sandrin; Filippo Romanato; Francesca Cecchinato; Anna Urciuolo; Dorianna Sandonà; Paolo De Coppi; Piero G Pavan; Martina Piccoli
Journal:  NPJ Regen Med       Date:  2022-04-25

5.  Assessing and enhancing migration of human myogenic progenitors using directed iPS cell differentiation and advanced tissue modelling.

Authors:  SungWoo Choi; Giulia Ferrari; Louise A Moyle; Kirsty Mackinlay; Naira Naouar; Salma Jalal; Sara Benedetti; Christine Wells; Francesco Muntoni; Francesco Saverio Tedesco
Journal:  EMBO Mol Med       Date:  2022-09-26       Impact factor: 14.260

6.  A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy.

Authors:  Neha R Dhoke; Hyunkee Kim; Sridhar Selvaraj; Karim Azzag; Haowen Zhou; Nelio A J Oliveira; Sudheer Tungtur; Carolina Ortiz-Cordero; James Kiley; Qi Long Lu; Anne G Bang; Rita C R Perlingeiro
Journal:  Cell Rep       Date:  2021-07-13       Impact factor: 9.423
  6 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.