Intracellular delivery by membrane disruption: mechanisms, strategies, and concepts

MP Stewart, R Langer, KF Jensen - Chemical reviews, 2018 - ACS Publications
Intracellular delivery is a key step in biological research and has enabled decades of
biomedical discoveries. It is also becoming increasingly important in industrial and medical …

Cellular dynamics in the muscle satellite cell niche

CF Bentzinger, YX Wang, NA Dumont, MA Rudnicki - EMBO reports, 2013 - embopress.org
Satellite cells, the quintessential skeletal muscle stem cells, reside in a specialized local
environment whose anatomy changes dynamically during tissue regeneration. The plasticity …

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy

DG Ousterout, AM Kabadi, PI Thakore… - Nature …, 2015 - nature.com
The CRISPR/Cas9 genome-editing platform is a promising technology to correct the genetic
basis of hereditary diseases. The versatility, efficiency and multiplexing capabilities of the …

Transplantation of genetically corrected human iPSC-derived progenitors in mice with limb-girdle muscular dystrophy

FS Tedesco, MFM Gerli, L Perani, S Benedetti… - Science translational …, 2012 - science.org
Mesoangioblasts are stem/progenitor cells derived from a subset of pericytes found in
muscle that express alkaline phosphatase. They have been shown to ameliorate the …

Stem cell therapy for muscular dystrophies

S Biressi, A Filareto, TA Rando - The Journal of Clinical …, 2020 - Am Soc Clin Investig
Muscular dystrophies are a heterogeneous group of genetic diseases, characterized by
progressive degeneration of skeletal and cardiac muscle. Despite the intense investigation …

Футурология

АВ Турчин - 2013 - elibrary.ru
В книге популярно излагаются методы футурологии и результаты, достигаемые
благодаря этим методам. Основная идея книги заключается в том, что главным …

Intra‐arterial transplantation of HLA‐matched donor mesoangioblasts in Duchenne muscular dystrophy

G Cossu, SC Previtali, S Napolitano… - EMBO molecular …, 2015 - embopress.org
Intra‐arterial transplantation of mesoangioblasts proved safe and partially efficacious in
preclinical models of muscular dystrophy. We now report the first‐in‐human, exploratory …

Engineered skeletal muscles for disease modeling and drug discovery

J Wang, A Khodabukus, L Rao, K Vandusen… - Biomaterials, 2019 - Elsevier
Skeletal muscle is the largest organ of human body with several important roles in everyday
movement and metabolic homeostasis. The limited ability of small animal models of muscle …

Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future

A Łoboda, J Dulak - Pharmacological Reports, 2020 - Springer
Background Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular
childhood disorder that causes progressive muscle weakness and degeneration and results …

3D human induced pluripotent stem cell–derived bioengineered skeletal muscles for tissue, disease and therapy modeling

L Pinton, M Khedr, VM Lionello, S Sarcar… - Nature …, 2023 - nature.com
Skeletal muscle is a complex tissue composed of multinucleated myofibers responsible for
force generation that are supported by multiple cell types. Many severe and lethal disorders …