Animal classification. by by J. E. Webb and J. H. Elgood.

By by J. E. Webb and J. H. Elgood.

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Manfredsson Conclusions The use of gene therapy to study, model, and treat neurological disorders has become increasingly popular in the last decade. What was previously envisioned as a futuristic and esoteric tool used by only a few research groups around the world, is now one of the more prevalent methods to perform genetic manipulations and target validation in vivo. However, it is often the case that data generated using gene therapy can be difficult to reproduce between groups. This is not because the results are not true.

30 Matthew L. Hirsch et al. Then, add a canonical intron donor sequence immediately following the vector A ORF (Fig. 2). Any sequence between this donor signal and the vector A 3′ ITR will be intronic (Fig. 2). To generate vector B, an intronic sequence is positioned downstream of the 5′ ITR and terminated by an intron acceptor sequence (Fig. 2). Then, the remainder of the ORF (prematurely truncated in vector A is positioned upstream of a poly-A sequence prior to the 3′ ITR of vector B (Fig. 2)).

Testa F et al (2013) Three-year follow-up after unilateral subretinal delivery of adenoassociated virus in patients with Leber congenital Amaurosis type 2. Ophthalmology 120(6): 1283–1291 20. Inagaki K et al (2007) The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice. J Virol 81(20):11304–11321 21. Choi VW, McCarty DM, Samulski RJ (2006) Host cell DNA repair pathways in adeno-associated viral genome processing. J Virol 80(21):10346–10356 22. Choi VW, Samulski RJ, McCarty DM (2005) Effects of adeno-associated virus DNA hairpin structure on recombination.

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