Pompe disease is an inherited neuromuscular disorder caused by deficiency of the
lysosomal enzyme acid alpha-glucosidase (GAA). The most severe form is infantile-onset …

The identification of patients with monogenic gene defects have illuminated the function of
different proteins in the immune system, including proteins that regulate the actin …

Wiskott-Aldrich syndrome (WAS) is a rare X-linked disorder characterized by combined
immunodeficiency, eczema, microthrombocytopenia, autoimmunity, and lymphoid …

Chimeric antigen receptor (CAR) T-cell therapies have revolutionised the field of
haematological malignancies by achieving impressive remission rates in patients with highly …

Gene therapy for the treatment of patients with Pompe disease is feasible and offers an
opportunity to fully correct the principal pathology leading to cellular glycogen accumulation …

As HSC GT is becoming available for more diseases, novel developments should focus on
improving availability while reducing costs of the treatment. Continued follow up of treated …

Primary immunodeficiencies (PIDs) are a group of rare inherited disorders of the immune
system. Many PIDs are devastating and require a definitive therapy to prevent progressive …

Three gene therapy strategies have received US Food and Drug Administration (FDA)
approval; one includes HIV-1-based lentiviral vectors. These vectors incorporate features to …

Inborn errors of immunity (IEIs) are a heterogeneous group of inherited disorders of the
immune system. Many IEIs have a severe clinical phenotype that results in progressive …

Lentiviral vectors (LVs) developed in the past two decades for research and pre-clinical
purposes have entered clinical trials with remarkable safety and efficacy performances …