First in man: gene editing for the treatment of transthyretin amyloidosis

被引:0
|
作者
Liuzzo, Giovanna [1 ]
Patrono, Carlo [2 ]
机构
[1] Catholic Univ, Sch Med, Fdn Policlin Univ A Gemelli IRCCS, Dept Cardiovasc & Pulm Sci, Largo A Gemelli 8, I-00168 Rome, Italy
[2] Catholic Univ, Sch Med, Fdn Policlin Univ A Gemelli IRCCS, Dept Pharmacol, Largo A Gemelli 8, I-00168 Rome, Italy
关键词
D O I
10.1093/eurheartj/ehab542
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
引用
收藏
页码:3597 / 3598
页数:2
相关论文
共 50 条
  • [41] Transthyretin amyloidosis
    Benson, MD
    Uemichi, T
    AMYLOID-INTERNATIONAL JOURNAL OF EXPERIMENTAL AND CLINICAL INVESTIGATION, 1996, 3 (01): : 44 - 56
  • [42] DEFINING TRANSTHYRETIN AMYLOIDOSIS PHENOTYPES IN THAOS : THE TRANSTHYRETIN AMYLOIDOSIS SURVEY
    Maurer, Mathew S.
    Plante-Bordeneuve, Violaine
    Coelho, Teresa
    Mundayat, Rajiv
    Karayal, Onur
    Rapezzi, Claudio
    JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY, 2013, 61 (10) : E1242 - E1242
  • [43] CLINICAL CHARACTERISTICS OF PATIENTS WITH TRANSTHYRETIN GENE MUTATIONS AND POLYNEUROPATHY MANIFESTATIONS OF HEREDITARY TRANSTHYRETIN AMYLOIDOSIS
    Khella, Sami
    Shah, Keyur
    Delgado, Diego
    Marti, Catherine
    Keller, Andrew
    Jefferies, John
    Towne, Meghan
    Gabriel, Aaron
    Narayana, Arvind
    Olugemo, Kemi
    MUSCLE & NERVE, 2021, 64 : S67 - S67
  • [44] Spectrum of transthyretin gene mutations and clinical characteristics of Polish patients with cardiac transthyretin amyloidosis
    Gawor, Monika
    Holcman, Katarzyna
    Franaszczyk, Maria
    Lipowska, Marta
    Michalek, Piotr
    Teresinska, Anna
    Bilinska, Zofia T.
    Rubis, Pawel
    Kostkiewicz, Magdalena
    Szot, Wojciech
    Podolec, Piotr
    Grzybowski, Jacek
    CARDIOLOGY JOURNAL, 2020, : 985 - 993
  • [45] Gene Editing as the Future of Cardiac Amyloidosis Therapeutics
    Sethi, Yashendra
    Mahtani, Arun Umesh
    Khehra, Nimrat
    Padda, Inderbir
    Patel, Neil
    Sebastian, Sneha Annie
    Malhi, Gurnaaz
    Kaiwan, Oroshay
    Saith, Sunil
    Johal, Gurpreet
    CURRENT PROBLEMS IN CARDIOLOGY, 2023, 48 (08)
  • [46] Clinical characteristics of patients with transthyretin gene mutations and polyneuropathy manifestations of hereditary transthyretin amyloidosis
    Khella, Sami
    Shah, Keyur
    Delgado, Diego
    Marti, Catherine
    Keller, Andrew
    Jefferies, John
    Towne, Meghan
    Gabriel, Aaron
    Narayana, Arvind
    Olugemo, Kemi
    JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM, 2021, 26 (03) : 390 - 391
  • [47] Imaging modalities in early cardiac transthyretin amyloidosis: who is first?
    Ney, Svenja
    Pennig, Lenhard
    Fassbach, Michael
    Hohmann, Christopher
    Pfister, Roman
    CLINICAL RESEARCH IN CARDIOLOGY, 2023, 112 (03) : 450 - 453
  • [48] Cardiac amyloidosis: diagnostic challenges and recent advancement in the treatment of transthyretin amyloidosis (ATTR)
    Rahman, Tanvir
    Moghadam, Reihaneh C.
    Agarwal, Vikram V.
    Reiss, Craig K.
    OXFORD MEDICAL CASE REPORTS, 2021, (08): : 283 - 287
  • [49] Diagnosis and treatment of transthyretin-related amyloidosis cardiomyopathy
    Teng, Catherine
    Li, Pengyang
    Bae, Ju Young
    Pan, Su
    Dixon, Richard A. F.
    Liu, Qi
    CLINICAL CARDIOLOGY, 2020, 43 (11) : 1223 - 1231
  • [50] Diagnosis and treatment of heart failure in hereditary transthyretin amyloidosis
    Gisela D. Puig-Carrion
    Alex Reyentovich
    Stuart D. Katz
    Clinical Autonomic Research, 2019, 29 : 45 - 53