Current Gene Therapy - Volume 18, Issue 6, 2018

Diamond Blackfan Anemia (DBA) is an inherited erythroid aplasia with onset in childhood. Patients carry heterozygous mutations in one of 19 Ribosomal Protein (RP) genes, that lead to defective ribosome biogenesis and function. Standard treatments include steroids or blood transfusions but the only definitive cure is allogeneic Hematopoietic Stem Cell Transplantation (HSCT). Although advances in HSCT have greatly impr Read More

Exosomes play a critical role in intercellular communication between cancer cells and their environments. These secreted nanovesicles can transfer different cargos such as mRNAs, proteins and microRNA (miRNA) to recipient cells. Exosomal miRNAs (exomiRs) derived from tumor cells have emerged as key players in cancer promotion via impairment of the immune system response, tumor growth, metastasis, angiogen Read More

Erectile Dysfunction (ED) is one of the most common conditions affecting middle-aged and older men. Over the past few decades, oral phosphodiesterase type 5 inhibitors have been used to treat ED. However, these oral medications require on-demand access and are not effective in some hard-totreat populations. Moreover, there are no effective alternative treatments for ED. Based on results from preclinical mod Read More

Introduction: Oncolytic virus therapy is currently considered as a promising therapeutic approach for cancer treatment. Adenovirus is well-known and extensively characterized as an oncolytic agent. The increasing number of clinical trials using this virus generates the demand for the development of a well-established purification approach. Triton X-100 is commonly used in cell lysis buffer preparations. The addition of Read More

Objective: Immunotherapy based on induction of T-cell responses is a promising approach to cancer treatment. The study aims to design artificial epitope-based immunogens, DNA vaccine candidates against melanoma and evaluate their ability to stimulate tumor cytotoxicity of ex vivo generated T-cells. Methods: The original computational methods were used for predicting T-cell epitopes and designing polyepitope mel Read More

Background: Leber's hereditary optic neuropathy is a hereditary mitochondrial disease. No effective treatment has so far been established, with gene therapy currently being the most promising. Because of the possibility of spontaneous visual acuity recovery in this disease, we screened patients before gene therapy, excluding those with spontaneous visual acuity improvement, and prepared for the subsequent gene ther Read More