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      Genetic Modification of the Lung Directed Toward Treatment of Human Disease

      1 , 1 , 1 , 1
      Human Gene Therapy
      Mary Ann Liebert Inc

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          RNA interference is mediated by 21- and 22-nucleotide RNAs.

          Double-stranded RNA (dsRNA) induces sequence-specific posttranscriptional gene silencing in many organisms by a process known as RNA interference (RNAi). Using a Drosophila in vitro system, we demonstrate that 21- and 22-nt RNA fragments are the sequence-specific mediators of RNAi. The short interfering RNAs (siRNAs) are generated by an RNase III-like processing reaction from long dsRNA. Chemically synthesized siRNA duplexes with overhanging 3' ends mediate efficient target RNA cleavage in the lysate, and the cleavage site is located near the center of the region spanned by the guiding siRNA. Furthermore, we provide evidence that the direction of dsRNA processing determines whether sense or antisense target RNA can be cleaved by the siRNA-protein complex.
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            Global burden of asthma among children.

            About 334 million people worldwide suffer from asthma, and this figure may be an underestimation. It is the most common chronic disease in children. Asthma is among the top 20 chronic conditions for global ranking of disability-adjusted life years in children; in the mid-childhood ages 5-14 years it is among the top 10 causes. Death rates from asthma in children globally range from 0.0 to 0.7 per 100 000. There are striking global variations in the prevalence of asthma symptoms (wheeze in the past 12 months) in children, with up to 13-fold differences between countries. Although asthma symptoms are more common in many high-income countries (HICs), some low- and middle-income countries (LMICs) also have high levels of asthma symptom prevalence. The highest prevalence of symptoms of severe asthma among children with wheeze in the past 12 months is found in LMICs and not HICs. From the 1990s to the 2000s, asthma symptoms became more common in some high-prevalence centres in HICs; in many cases, the prevalence stayed the same or even decreased. At the same time, many LMICs with large populations showed increases in prevalence, suggesting that the overall world burden is increasing, and that therefore global disparities in asthma prevalence are decreasing. The costs of asthma, where they have been estimated, are relatively high. The global burden of asthma in children, including costs, needs ongoing monitoring using standardised methods.
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              Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics.

              Considered by some to be among the simpler forms of life, viruses represent highly evolved natural vectors for the transfer of foreign genetic information into cells. This attribute has led to extensive attempts to engineer recombinant viral vectors for the delivery of therapeutic genes into diseased tissues. While substantial progress has been made, and some clinical successes are over the horizon, further vector refinement and/or development is required before gene therapy will become standard care for any individual disorder.
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                Author and article information

                Journal
                Human Gene Therapy
                Human Gene Therapy
                Mary Ann Liebert Inc
                1043-0342
                1557-7422
                January 2017
                January 2017
                : 28
                : 1
                : 3-84
                Affiliations
                [1 ]Department of Genetic Medicine, Weill Cornell Medical College, New York, New York.
                Article
                10.1089/hum.2016.152
                27927014
                978acad9-1f1e-4572-b31e-d8d53fc7c308
                © 2017

                http://www.liebertpub.com/nv/resources-tools/text-and-data-mining-policy/121/

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