2015 The Neuroscientist : a review journal bringing neurobiology, neurology and psychiatry
* Adeno-associated virus vectors and neurological gene therapy.
- Gene therapy has strong potential for treating a variety of genetic disorders, as demonstrated in recent clinical trials. There is unfortunately no scarcity of disease targets, and the grand challenge in this field has instead been the development of safe and efficient gene delivery platforms. To date, approximately two thirds of the 1800 gene therapy clinical trials completed worldwide have used viral vectors. Among these, adeno-associated virus (AAV) has emerged as particularly promising because of its impressive safety profile and efficiency in transducing a wide range of cell types. Gene delivery to the CNS involves both considerable promise and unique challenges, and better AAV vectors are thus needed to translate CNS gene therapy approaches to the clinic. This review discusses strategies for vector design, potential routes of administration, immune responses, and clinical applications of AAV in the CNS.
Overview of verb demonstrate
The verb demonstrate has 4 senses (first 4 from tagged texts)
1. (24) show, demo, exhibit, present, demonstrate -- (give an exhibition of to an interested
audience; "She shows her dogs frequently"; "We will demo the new software in Washington")
2. (22) prove, demonstrate, establish, show, shew -- (establish the validity of something, as by an
example, explanation or experiment; "The experiment demonstrated the instability of the compound";
"The mathematician showed the validity of the conjecture")
3. (2) attest, certify, manifest, demonstrate, evidence -- (provide evidence for; stand as proof of;
show by one's behavior, attitude, or external attributes; "His high fever attested to his illness";
"The buildings in Rome manifest a high level of architectural sophistication"; "This decision
demonstrates his sense of fairness")
4. (1) demonstrate, march -- (march in protest; take part in a demonstration; "Thousands
demonstrated against globalization during the meeting of the most powerful economic nations in
Seattle")
Overview of adj demonstrated
The adj demonstrated has 1 sense (first 1 from tagged texts)
1. (1) demonstrated -- (having been demonstrated or verified beyond doubt)
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