Lentiviral Vector-Based Gene Therapy in Parkinson Disease

Beta thalassemia gene therapy using lentiviral vectors

Recent years, allogeneic bone marrow transplantation (BMT) has proved to be the successful cure for patients with thalassemia major, however this is restricted due to limited matched-related donor. Its complications include chronic graft-versus-host disease in 5-8% of patients. So, a molecular approach, such as gene therapy for direct normal beta globin gene transmission, seems quite promising ...

Lentiviral vector-based therapy in head and neck cancer (Review)

Head and neck squamous cell carcinoma (HNSCC) is the sixth most common neoplasm worldwide. Despite advances in multimodality treatments involving surgery, radiation and chemotherapy, the five-year survival rate has remained at ~50% for the past 35 years. Therefore, the early detection of recurrent or persistent disease is extremely important. Replication-incompetent HIV-1-based lentiviral vecto...

Neuroprotection in the rat Parkinson model by intrastriatal GDNF gene transfer using a lentiviral vector.

We used a recombinant lentiviral vector (rLV) for gene delivery of GDNF to the striatum, and assessed its neuroprotective effects in the intrastriatal 6-hydroxydopamine (6-OHDA) lesion model. The level of GDNF expression obtained with the rLV-GDNF vector was dose-related and ranged between 0.9-9.3 ng/mg tissue in the transduced striatum, as determined by ELISA, and 0.2-3.0 ng/mg tissue were det...

Gene Regulatable Lentiviral Vector System

Lentiviral-mediated gene therapy for bone disease in MPS

Introduction: Mucopolysaccharidoses (MPS) are a group of 11 inherited metabolic disorders arising from a deficiency in a lysosomal protein required for the degradation of glycosaminoglycan (gag) carbohydrates. MPS type VII (MPS VII) is an autosomal recessive lysosomal storage disorder caused by β-glucuronidase deficiency resulting in the accumulation of chondroitin sulphate, heparan sulphate an...