siRNA and the lung: research tool or therapeutic drug?

被引:40
作者
de Fougerolles, Antonin [1 ]
Novobrantseva, Tatiana [1 ]
机构
[1] Alnylam Pharmaceut, Cambridge, MA 02142 USA
关键词
D O I
10.1016/j.coph.2008.04.005
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Oligonucleotide-based therapeutics have been hailed as 'the next great wave of the biotechnology revolution' starting with antisense oligonucleotides (ASOs) nearly 20 years ago to RNA interference (RNAi) currently. Is RNAi just the latest research tool or does it have real potential as a therapeutic drug modality? As a research tool, it is evident that RNAi has revolutionized the biological sciences by allowing selective silencing of messenger RNA (mRNA) expression. With the advent of the postgenomic era, RNAi offers a therapeutic platform on which to identify potential picomolar active drug candidates to any target, including those that are conventionally undruggable. In this review, we will discuss the progress made in developing RNAi therapeutics for the treatment of respiratory diseases.
引用
收藏
页码:280 / 285
页数:6
相关论文
共 31 条
[1]   RNAi therapeutics: Principles, prospects and challenges [J].
Aagaard, Lars ;
Rossi, John J. .
ADVANCED DRUG DELIVERY REVIEWS, 2007, 59 (2-3) :75-86
[2]   Hyperoxia causes angiopoietin 2-mediated acute lung injury and necrotic cell death [J].
Bhandari, Vineet ;
Choo-Wing, Rayman ;
Lee, Chun G. ;
Zhu, Zhou ;
Nedrelow, Jonathan H. ;
Chupp, Geoffrey L. ;
Zhang, Xucher ;
Matthay, Michael A. ;
Ware, Lorraine B. ;
Homer, Robert J. ;
Lee, Patty J. ;
Geick, Anke ;
de Fougerolles, Antonin R. ;
Elias, Jack A. .
NATURE MEDICINE, 2006, 12 (11) :1286-1293
[3]   Pulmonary delivery of nucleic acids [J].
Birchall, James .
EXPERT OPINION ON DRUG DELIVERY, 2007, 4 (06) :575-578
[4]   Inhibition of respiratory viruses by nasally administered siRNA [J].
Bitko, V ;
Musiyenko, A ;
Shulyayeva, O ;
Barik, S .
NATURE MEDICINE, 2005, 11 (01) :50-55
[5]   RNAi therapeutics: a potential new class of pharmaceutical drugs [J].
Bumcrot, David ;
Manoharan, Muthiah ;
Koteliansky, Victor ;
Sah, Dinah W. Y. .
NATURE CHEMICAL BIOLOGY, 2006, 2 (12) :711-719
[6]   Interfering with disease: a progress report on siRNA-based therapeutics [J].
de Fougerolles, Antonin ;
Vornlocher, Hans-Peter ;
Maraganore, John ;
Lieberman, Judy .
NATURE REVIEWS DRUG DISCOVERY, 2007, 6 (06) :443-453
[7]   Delivery vehicles for small interfering RNA in vivo [J].
De Fougerolles, Antonin R. .
HUMAN GENE THERAPY, 2008, 19 (02) :125-132
[8]   Reconstituted influenza virus envelopes as an efficient carrier system for cellular delivery of small-interfering RNAs [J].
de Jonge, J ;
Holtrop, M ;
Wilschut, J ;
Huckriede, A .
GENE THERAPY, 2006, 13 (05) :400-411
[9]   Inhibition of influenza virus production in virus-infected mice by RNA interference [J].
Ge, Q ;
Filip, L ;
Bai, AL ;
Nguyen, T ;
Eisen, HN ;
Chen, J .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2004, 101 (23) :8676-8681
[10]   Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo [J].
Griesenbach, U ;
Kitson, C ;
Garcia, SE ;
Farley, R ;
Singh, C ;
Somerton, L ;
Painter, H ;
Smith, RL ;
Gill, DR ;
Hyde, SC ;
Chow, YH ;
Hu, J ;
Gray, M ;
Edbrooke, M ;
Ogilvie, V ;
MacGregor, G ;
Scheule, RK ;
Cheng, SH ;
Caplen, NJ ;
Alton, EWFW .
RESPIRATORY RESEARCH, 2006, 7 (1)