منابع مشابه
Gene delivery to the retina using lentiviral vectors.
The delivery of foreign DNA to the retina has proven to be a valuable tool for investigations of retinal disease, development, and complex cellular interactions. To achieve efficient and stable retinal gene expression with minimal unwanted side effects, viral vectors derived from AAV (adeno-associated virus) and LV (lentivirus) remain the vehicles of choice. LV vectors have gained recent attent...
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The present study aimed to evaluate the incorporation of protamine into niosome/DNA vectors to analyze the potential application of this novel ternary formulation to deliver the pCMS-EGFP plasmid into the rat retina. Binary vectors based on niosome/DNA and ternary vectors based on protamine/DNA/niosomes were prepared and physicochemically characterized. In vitro experiments were performed in AR...
متن کاملVectors for Gene Delivery
Gene therapy bases its rationale on the transfer of genetic components (genes or fragments thereof) into somatic cells, with the aim of preventing, correcting, or healing various types of disorders. After this introductory sentence you expect us to start telling you some of the marvellous achievements in setting up the tools that allow this transfer. However, before entering into the intricate ...
متن کاملTargeted synthetic gene delivery vectors.
Synthetic gene delivery vehicles have made significant progress in the past decade in demonstrating strong potential for targeted delivery to specific cells, low toxicity and immunogenicity and large carrying capacity. However, significant advances must still be made to increase the efficiency of both polymer and lipid vehicles. Furthermore, techniques to generate more effective targeting moiet...
متن کاملEffective delivery of large genes to the retina by dual AAV vectors
Retinal gene therapy with adeno-associated viral (AAV) vectors is safe and effective in humans. However, AAV's limited cargo capacity prevents its application to therapies of inherited retinal diseases due to mutations of genes over 5 kb, like Stargardt's disease (STGD) and Usher syndrome type IB (USH1B). Previous methods based on 'forced' packaging of large genes into AAV capsids may not be ea...
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ژورنال
عنوان ژورنال: Annual Review of Vision Science
سال: 2017
ISSN: 2374-4642,2374-4650
DOI: 10.1146/annurev-vision-102016-061413