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  2. Volume 7, Issue 8
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Online ISSN: 2515-8260

Volume7, Issue8

GENE THERAPY for the TREATMENT of X-LINKED RETINITIS PIGMENTOSA: A REVIEW

    Azhar Dzulhadj B. Arafah, Burhanuddin Arafah, Azhariah Nur B. Arafah

European Journal of Molecular & Clinical Medicine, 2020, Volume 7, Issue 8, Pages 1524-1532

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Abstract

Gene therapy is a medical procedure that inserts a normal sequence of genetic material into living cells to rectify a malfunctioning DNA by replacing, altering or augmenting the defective form of the DNA. Gene Therapy has been known to be a promising treatment for genetic disorders, especially monogenic diseases such as Inherited Retinal Disorders (IRD). One of the most common types of IRD is X-Linked Retinitis Pigmentosa (XLRP), which is attributed to the mutation of the retinitis pigmentosa GTPase regulatore (RPGR) gene.  Gene therapy using engineered CRISPR-Cas9 complex and Adeno-associated viral (AAV) vectors is capable to detect DNA mutation which could lead to an efficient transfection. This article reviews the underlying molecular mechanism of XLRP; the characteristics of the blood-retinal barrier (BRB) and other cellular structures of the retina as the ideal site to perform gene delivery; as well as recommended approaches in performing subretinal injection to achieve optimal outcomes.
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(2020). GENE THERAPY for the TREATMENT of X-LINKED RETINITIS PIGMENTOSA: A REVIEW. European Journal of Molecular & Clinical Medicine, 7(8), 1524-1532.
Azhar Dzulhadj B. Arafah, Burhanuddin Arafah, Azhariah Nur B. Arafah. "GENE THERAPY for the TREATMENT of X-LINKED RETINITIS PIGMENTOSA: A REVIEW". European Journal of Molecular & Clinical Medicine, 7, 8, 2020, 1524-1532.
(2020). 'GENE THERAPY for the TREATMENT of X-LINKED RETINITIS PIGMENTOSA: A REVIEW', European Journal of Molecular & Clinical Medicine, 7(8), pp. 1524-1532.
GENE THERAPY for the TREATMENT of X-LINKED RETINITIS PIGMENTOSA: A REVIEW. European Journal of Molecular & Clinical Medicine, 2020; 7(8): 1524-1532.
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