Targeting the myostatin signaling pathway to treat muscle loss and metabolic dysfunction

SJ Lee - The Journal of clinical investigation, 2021 - Am Soc Clin Investig
Since the discovery of myostatin (MSTN; also known as GDF-8) as a critical regulator of
skeletal muscle mass in 1997, there has been an extensive effort directed at understanding …

[HTML][HTML] Sarcopenia: its assessment, etiology, pathogenesis, consequences and future perspectives

Y Rolland, S Czerwinski, GA Van Kan, JE Morley… - The Journal of nutrition …, 2008 - Elsevier
Sarcopenia is a loss of muscle protein mass and loss of muscle function. It occurs with
increasing age, being a major component in the development of frailty. Current knowledge …

[PDF][PDF] In vivo target gene activation via CRISPR/Cas9-mediated trans-epigenetic modulation

HK Liao, F Hatanaka, T Araoka, P Reddy, MZ Wu, Y Sui… - Cell, 2017 - cell.com
Current genome-editing systems generally rely on inducing DNA double-strand breaks
(DSBs). This may limit their utility in clinical therapies, as unwanted mutations caused by …

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

KD Foust, E Nurre, CL Montgomery, A Hernandez… - Nature …, 2009 - nature.com
Delivery of genes to the brain and spinal cord across the blood-brain barrier (BBB) has not
yet been achieved. Here we show that adeno-associated virus (AAV) 9 injected …

Uraemic sarcopenia: aetiology and implications

IH Fahal - Nephrology Dialysis Transplantation, 2014 - academic.oup.com
The term uraemic myopathy has been used loosely to describe the skeletal muscle
abnormalities in uraemic patients. However, it does not fully explain the observed …

The pathogenesis and therapy of muscular dystrophies

S Guiraud, A Aartsma-Rus, NM Vieira… - Annual review of …, 2015 - annualreviews.org
Current molecular genomic approaches to human genetic disorders have led to an
explosion in the identification of the genes and their encoded proteins responsible for these …

A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy

JR Mendell, Z Sahenk, V Malik, AM Gomez… - Molecular Therapy, 2015 - cell.com
Becker muscular dystrophy (BMD) is a variant of dystrophin deficiency resulting from DMD
gene mutations. Phenotype is variable with loss of ambulation in late teenage or late mid-life …

Myostatin: a skeletal muscle chalone

SJ Lee - Annual review of physiology, 2023 - annualreviews.org
Myostatin (GDF-8) was discovered 25 years ago as a new transforming growth factor-β
family member that acts as a master regulator of skeletal muscle mass. Myostatin is made by …

Role of skeletal muscle in insulin resistance and glucose uptake

KE Merz, DC Thurmond - Comprehensive Physiology, 2011 - Wiley Online Library
The skeletal muscle is the largest organ in the body, by mass. It is also the regulator of
glucose homeostasis, responsible for 80% of postprandial glucose uptake from the …

Regulation of muscle mass by follistatin and activins

SJ Lee, YS Lee, TA Zimmers, A Soleimani… - Molecular …, 2010 - academic.oup.com
Myostatin is a TGF-β family member that normally acts to limit skeletal muscle mass.
Follistatin is a myostatin-binding protein that can inhibit myostatin activity in vitro and …