Clinical features of Danon disease and insights gained from LAMP-2 deficiency models
Review article
[키워드] absence
animal model
autophagic
cardiac
cells
characterized
clinical
clinical feature
clinical trial
contribute
deficiency
demonstrated
disease
disorder
drug
dysfunction
Effect
effective
Evidence
feature
Gene therapy
humans
hypertrophic cardiomyopathy
impairment
in vitro
in vivo
isoform
LAMP2
mechanism
Mental
mimicking
molecular
Mutation
pathology
Patient
phenotype
pluripotent stem cell
potential therapy
powerful tool
Protein
provided
Research
resulting
Skeletal muscle
Symptom
therapeutic
Treatment
weakness
while
X-linked
[DOI] 10.1016/j.tcm.2021.10.012 [Article Type] Review article
[DOI] 10.1016/j.tcm.2021.10.012 [Article Type] Review article