Remarkable advancements have been made in understanding the pathogenesis of
Alzheimer's disease, Parkinson's disease, and other neurological disease; in our depth of …

The transformative potential of gene therapy has been demonstrated in humans. However,
there is an unmet need for non-invasive targeted gene delivery and regulation in the …

Human embryonic stem cell (hESC)-derived midbrain dopaminergic (mDA) cell
transplantation is a promising therapeutic strategy for Parkinson's disease (PD). Here, we …

Mucopolysaccharidoses are inherited metabolic disorders caused by the deficiency in
lysosomal enzymes required to break down glycosaminoglycans. Accumulation of …

Few models allow the study of neurite damage in the human central nervous system. We
used here dopaminergic LUHMES neurons to establish a culture system that allows for (i) …

Background: The development and use of human embryonic stem cells (hESCs) in
regenerative medicine have been revolutionary, offering significant advancements in …

In vitro-generated pluripotent stem cell (PSC)-derived Pax3-induced (iPax3) myogenic
progenitors display an embryonic transcriptional signature, but upon engraftment, the profile …

Rapid advances in biological knowledge and technological innovation have greatly
advanced the fields of stem cell and gene therapies to combat a broad spectrum of …

Parkinson's disease (PD) is characterized by the loss of the dopaminergic nigrostriatal
pathway which has led to the successful development of drug therapies that replace or …

Parkinson's disease (PD) is a neurodegenerative condition characterized by the loss of
midbrain dopaminergic neurons, leading to motor symptoms. While current treatments …