Gastrointestinal stromal tumours

JY Blay, YK Kang, T Nishida… - Nature reviews Disease …, 2021 - nature.com
Gastrointestinal stromal tumours (GIST) have an incidence of~ 1.2 per 105 individuals per
year in most countries. Around 80% of GIST have varying molecular changes, predominantly …

Therapeutic approaches for Duchenne muscular dystrophy

TC Roberts, MJA Wood, KE Davies - Nature Reviews Drug Discovery, 2023 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

Pan-cancer whole-genome analyses of metastatic solid tumours

P Priestley, J Baber, MP Lolkema, N Steeghs… - Nature, 2019 - nature.com
Metastatic cancer is a major cause of death and is associated with poor treatment efficacy. A
better understanding of the characteristics of late-stage cancer is required to help adapt …

Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division

NA Dumont, YX Wang, J Von Maltzahn, A Pasut… - Nature medicine, 2015 - nature.com
Dystrophin is expressed in differentiated myofibers, in which it is required for sarcolemmal
integrity, and loss-of-function mutations in the gene that encodes it result in Duchenne …

Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

JW McGreevy, CH Hakim… - Disease models & …, 2015 - journals.biologists.com
Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

In silico prescription of anticancer drugs to cohorts of 28 tumor types reveals targeting opportunities

C Rubio-Perez, D Tamborero, MP Schroeder… - Cancer cell, 2015 - cell.com
Large efforts dedicated to detect somatic alterations across tumor genomes/exomes are
expected to produce significant improvements in precision cancer medicine. However, high …

Gastrointestinal stromal tumor: challenges and opportunities for a new decade

C Serrano, S George - Clinical Cancer Research, 2020 - AACR
Gastrointestinal stromal tumor (GIST) provides a paradigm to evaluate new molecularly
targeted therapies and to identify structural and functional mechanisms for drug response …

[HTML][HTML] The GIST of advances in treatment of advanced gastrointestinal stromal tumor

IM Schaefer, RP DeMatteo… - American Society of …, 2022 - ncbi.nlm.nih.gov
Gastrointestinal stromal tumor (GIST) is the most common malignant neoplasm of
mesenchymal origin and a compelling clinical and biologic model for the rational …

Complementary activity of tyrosine kinase inhibitors against secondary kit mutations in imatinib-resistant gastrointestinal stromal tumours

C Serrano, A Mariño-Enríquez, DL Tao, J Ketzer… - British journal of …, 2019 - nature.com
Background Most patients with KIT-mutant gastrointestinal stromal tumours (GISTs) benefit
from imatinib, but treatment resistance results from outgrowth of heterogeneous subclones …

Satellite cells in muscular dystrophy–lost in polarity

NC Chang, FP Chevalier, MA Rudnicki - Trends in molecular medicine, 2016 - cell.com
Recent findings employing the mdx mouse model for Duchenne muscular dystrophy (DMD)
have revealed that muscle satellite stem cells play a direct role in contributing to disease …