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Recent progress in Gene Therapy and Other Targeted Therapeutic Approaches for Beta Thalassemia.
Abstract Thalassemia syndromes are a group of inherited blood disorders caused by limitation or absence of alpha or beta- globin chain synthesis resulting in ineffective erythropoiesis and chronic hemolytic anemia. According to the clinical fact of thalassemia as recessive autosomal, thalassemia divided into alpha and beta thalassemia. The main complication of beta thalassemia is excessive red blood cells destruction, oxidative stress, extramedullary hematopoiesis pseudotumors, hemosiderosis-induced organ dysfunction, malignancy, polyneuropathy, myopathy, osteoporosis, and leg ulcers. Although, the first obligator...
Source: Current Drug Targets - July 25, 2019 Category: Drugs & Pharmacology Authors: Hamed EM, Meabed MH, Aly UF, Hussein RRS Tags: Curr Drug Targets Source Type: research

Full‐length dysferlin expression driven by engineered human dystrophic blood derived CD133+ stem cells
The protein dysferlin is abundantly expressed in skeletal and cardiac muscles, where its main function is membrane repair. Mutations in the dysferlin gene are involved in two autosomal recessive muscular dystrophies: Miyoshi myopathy and limb‐girdle muscular dystrophy type 2B. Development of effective therapies remains a great challenge. Strategies to repair the dysferlin gene by skipping mutated exons, using antisense oligonucleotides (AONs), may be suitable only for a subset of mutations, while cell and gene therapy can be extended to all mutations. AON‐treated blood‐derived CD133+ stem cells isolated from patients...
Source: FEBS Journal - October 8, 2013 Category: Research Authors: Mirella Meregalli, Claire Navarro, Clementina Sitzia, Andrea Farini, Erica Montani, Nicolas Wein, Paola Razini, Cyriaque Beley, Letizia Cassinelli, Daniele Parolini, Marzia Belicchi, Dario Parazzoli, Luis Garcia, Yvan Torrente Tags: Original Article Source Type: research

Full‐Length Dysferlin Expression Driven by Engineered Human Dystrophic Blood‐Derived CD133+ Stem Cells
This article is protected by copyright. All rights reserved.
Source: FEBS Journal - September 13, 2013 Category: Research Authors: Mirella Meregalli, Claire Navarro, Clementina Sitzia, Andrea Farini, Erica Montani, Nicolas Wein, Paola Razini, Cyriaque Beley, Letizia Cassinelli, Daniele Parolini, Marzia Belicchi, Dario Parazzoli, Luis Garcia, Yvan Torrente Tags: Original Article Source Type: research