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Review
. 2017 Jul;10(4):242-248.
doi: 10.1111/cts.12466. Epub 2017 May 23.

Gene Therapy 2017: Progress and Future Directions

Affiliations
Review

Gene Therapy 2017: Progress and Future Directions

A M Keeler et al. Clin Transl Sci. 2017 Jul.
No abstract available

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Figures

Figure 1
Figure 1
Timeline of major events in clinical gene therapy. A few selected key dates in the history of human gene therapy are depicted, with the dates indicated on the x‐axis. AAV2, adeno‐associated virus type 2; Ad5, adenovirus type 5; CF, cystic fibrosis; CRISPR, clustered regularly interspaced short palindromic repeats; FIX, clotting factor IX; LPL, lipoprotein lipase; OTC, ornithine transcambamylase; RNAi, RNA inhibition; RPE65, 65 kilo‐Dalton retinal pigment epithelial protein; RV, gammaretrovirus; SCID‐ADA, severe combined immune deficiency due to adenosine deaminase deficiency; TIL, tumor‐infiltrating lymphocytes.
Figure 2
Figure 2
Structure of adeno‐associated virus 2 (AAV2).113 The icosahedral structure of the virus capsid is shown. Note that this structure is symmetrical across a twofold, threefold, and fivefold axis of symmetry. The ability of the AAV vectors to transduce various cell types largely depends on variation in amino acid moieties highlighted in color.

References

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