AD

PD

Dementia

Gene therapy can be used to treat diseases by the introduction of therapeutic genes, by replacing, silencing, or correcting faulty genes.

·         APOE is the genetic risk factor.

Significantly increases the incidence of late-onset AD (LOAD) [56,57].

·         ECE can participate in Aβ degradation and could be a potential target for a pharmacological or gene therapeutic approach [58,59].

·         Lentivirus mediated gene delivery of cathepsin B reduced anti-Aβ antibody, and Thioflavin S-positive amyloid plaques, supporting a role for cathepsin B in Aβ degradation [64].

·         Gene delivery of IDE could possibly enhance Aβ degradation and influence AD pathology [66,67].

·         Down-regulation of AD-associated proteins, BAC1, APP, by siRNA [66,67].

·         Genome editing of mutations in several genes associated with both familial- and sporadic PD, including parkin, LRRK2, SNCA, PINK1, DJ-1, VPS35, DNAJC13, CHCHD2 [59].

·         Gene therapy with growth factors and also some non-disease modifying targets, such as TH, GCH and AADC and VMAT2 could be relevant treatment for patients [60].

·         Approximately 30% of patients with FTD show a clear autosomal dominant inheritance pattern, where one copy of a mutated gene causes the genetic condition.

·         The three genes commonly involved in hereditary of FTD are granulin (GRN), C9orf72 and MAPT [61,62].

·         AAV gene therapy is a potential avenue for disease modification in GRN carriers [68,69].