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Comment
. 2021 Sep 1;29(9):2633-2634.
doi: 10.1016/j.ymthe.2021.08.014. Epub 2021 Aug 24.

In vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis

Ana Rita Batista  1 , Terence R Flotte  2
Affiliations
Comment

In vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis

Ana Rita Batista et al. Mol Ther. .
No abstract available

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Comment on

  • CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
    Gillmore JD, Gane E, Taubel J, Kao J, Fontana M, Maitland ML, Seitzer J, O'Connell D, Walsh KR, Wood K, Phillips J, Xu Y, Amaral A, Boyd AP, Cehelsky JE, McKee MD, Schiermeier A, Harari O, Murphy A, Kyratsous CA, Zambrowicz B, Soltys R, Gutstein DE, Leonard J, Sepp-Lorenzino L, Lebwohl D. Gillmore JD, et al. N Engl J Med. 2021 Aug 5;385(6):493-502. doi: 10.1056/NEJMoa2107454. Epub 2021 Jun 26. N Engl J Med. 2021. PMID: 34215024 Clinical Trial.

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