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222. Nexiguran Ziclumeran Gene Editing in Hereditary ATTR with Polyneuropathy.
作者: Julian D Gillmore.;Ed Gane.;Jörg Täubel.;Björn Pilebro.;Andoni Echaniz-Laguna.;Justin Kao.;William Litchy.;Safi Shahda.;Alexandra Haagensen.;Liron Walsh.;Derek Smith.;Jessica Kachadourian.;Jonathan H Ward.;David Lebwohl.;Peijuan Zhu.;Yuanxin Xu.;Adia Leung.;Alison Sonderfan.;David E Gutstein.;Garen Manvelian.;David Adams.
来源: N Engl J Med. 2025年393卷14期1375-1386页
Hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, multisystem, progressive, debilitating, and fatal disease characterized by tissue deposition of misfolded transthyretin (TTR) in peripheral nerves. Nexiguran ziclumeran (nex-z) is an investigational in vivo therapy based on CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats and associated Cas9 endonuclease) that is designed to reduce serum TTR levels through selective inactivation of TTR in the liver.
225. Permethrin-Treated Baby Wraps for the Prevention of Malaria.
作者: Ross M Boyce.;Bonnie E Shook-Sa.;Ronnie Ndizeye.;Emmanuel Baguma.;Dana Giandomenico.;Caitlin A Cassidy.;Solomon Eshun.;Mark J Siedner.;Sarah G Staedke.;Moses Ntaro.;Jonathan J Juliano.;Raquel Reyes.;Edgar M Mulogo.
来源: N Engl J Med. 2025年393卷14期1399-1408页
Malaria remains a major cause of childhood death in sub-Saharan Africa. We leveraged the traditional practice of mothers carrying children on their backs in cloth wraps to assess whether treating the wraps with an insect repellent might provide a layer of protection against malaria.
237. CD19 CAR T-Cell Therapy in Multidrug-Resistant Ulcerative Colitis.
作者: Fabian Müller.;Raja Atreya.;Simon Völkl.;Michael Aigner.;Sascha Kretschmann.;Soraya Kharboutli.;Moritz Leppkes.;Selina Sitte.;Deike Strobel.;Arndt Hartmann.;Saurabh Mehandru.;Jean-Frederic Colombel.;Georg Schett.;Andreas Mackensen.;Markus F Neurath.
来源: N Engl J Med. 2025年393卷12期1239-1241页 |