Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches
نویسندگان
چکیده
منابع مشابه
Ex vivo gene therapy for HIV-1 treatment.
Until recently, progress in ex vivo gene therapy (GT) for human immunodeficiency virus-1 (HIV-1) treatment has been incremental. Long-term HIV-1 remission in a patient who received a heterologous stem cell transplant for acquired immunodeficiency syndrome-related lymphoma from a CCR5(-/-) donor, even after discontinuation of conventional therapy, has energized the field. We review the status of...
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Background and Aim: Diabetes is a common disorder worldwide, and exhaustive efforts have been made to cure this disease. Gene therapy has considered as a potential curative method that has more stability in comparison with the other pharmaceutical methods. However, the application of gene therapy as a definitive treatment demands further investigation. This study aim is to prepare a suitable hi...
متن کاملDesigning an Engineered Construct Gene Sensitive to Carbohydrate In-vitro and Candidate for Human Insulin Gene Therapy In-vivo
Background and Aim: Diabetes is a common disorder worldwide, and exhaustive efforts have been made to cure this disease. Gene therapy has considered as a potential curative method that has more stability in comparison with the other pharmaceutical methods. However, the application of gene therapy as a definitive treatment demands further investigation. This study aim is to prepare a suitable hi...
متن کاملLinarin Inhibits the Acetylcholinesterase Activity In-vitro and Ex-vivo
Linarin is a flavone glycoside in the plants Flos chrysanthemi indici, Buddleja officinalis, Cirsium setosum, Mentha arvensis and Buddleja davidii, and has been reported to possess analgesic, antipyretic, anti-inflammatory and neuroprotective activities. In this paper, linarin was investigated for its AChE inhibitory potential both in vitro and ex vivo. Ellman’s colorimetric method was used for...
متن کاملAn ex vivo gene therapy approach in X-linked retinoschisis
PURPOSE X-linked retinoschisis (XLRS) is juvenile-onset macular degeneration caused by haploinsufficiency of the extracellular cell adhesion protein retinoschisin (RS1). RS1 mutations can lead to either a non-functional protein or the absence of protein secretion, and it has been established that extracellular deficiency of RS1 is the underlying cause of the phenotype. Therefore, we hypothesize...
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ژورنال
عنوان ژورنال: Italian Journal of Pediatrics
سال: 2018
ISSN: 1824-7288
DOI: 10.1186/s13052-018-0565-y