Several viral vector-based gene therapy drugs have now received marketing approval. A
much larger number of additional viral vectors are in various stages of clinical trials for the …

Loss-of-function heterozygous mutations in GRN, the gene encoding progranulin (PGRN),
were identified in patients with frontotemporal lobar degeneration (FTLD) almost two …

Frontotemporal dementia (FTD) is a highly heritable group of neurodegenerative disorders,
with around 30% of patients having a strong family history. The majority of that heritability is …

So far, gene therapies have relied on complex constructs that cannot be finely controlled,.
Here we report a universal switch element that enables precise control of gene replacement …

Hematopoietic stem cell transplantation can deliver therapeutic proteins to the central
nervous system (CNS) through transplant-derived microglia-like cells. However, current …

In recent years, gene therapy has been raising hopes toward viable treatment strategies for
rare genetic diseases for which there has been almost exclusively supportive treatment. We …

Focused ultrasound combined with intravenously injected microbubbles (FUS) is known to
non-invasively, locally, and transiently increase the permeability of the blood-brain barrier …

Neuronal ceroid lipofuscinosis (NCLs) is a group of inherited neurodegenerative lysosomal
storage diseases that together represent the most common cause of dementia in children …

Frontotemporal dementia (FTD) is a neurodegenerative disease, leading to behavioral
changes and language difficulties. Heterozygous loss-of-function mutations in progranulin …

Therapeutic viral gene delivery is an emerging technology which aims to correct genetic
mutations by introducing new genetic information to cells either to correct a faulty gene or to …