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Titolo:
Respirable antisense oligonucleotides
Autore:
Nyce, JW;
Indirizzi:
EpiGenesis Pharmaceut Inc, Princeton, NJ 08543 USA EpiGenesis Pharmaceut Inc Princeton NJ USA 08543 Princeton, NJ 08543 USA
Titolo Testata:
NEW DRUGS FOR ASTHMA, ALLERGY AND COPD
, volume: 31, anno: 2001,
pagine: 361 - 364
SICI:
1422-2140(2001)31:<361:RAO>2.0.ZU;2-A
Fonte:
ISI
Lingua:
ENG
Soggetto:
ADENOSINE-INDUCED BRONCHOCONSTRICTION; ALLERGIC RABBITS; II PNEUMOCYTES; RECEPTOR; SURFACTANT; THERAPEUTICS; EXPRESSION; ASTHMA; SYSTEM;
Tipo documento:
Article
Natura:
Collana
Settore Disciplinare:
Clinical Medicine
Citazioni:
21
Recensione:
Indirizzi per estratti:
Indirizzo: Nyce, JW EpiGenesis Pharmaceut Inc, Princeton, NJ 08543 USA EpiGenesis Pharmaceut Inc Princeton NJ USA 08543 n, NJ 08543 USA
Citazione:
J.W. Nyce, "Respirable antisense oligonucleotides", PROG R RES, 31, 2001, pp. 361-364

Abstract

Like diseases in other organ systems, virtually every major respiratory disease can be characterized by the discordant expression of one or more genes whose protein products contribute substantially to the underlying pathophysiology. Asthma perhaps exemplifies this process, where evidence has been obtained to support a role for more than 100 different candidate mediators. Respirable antisense oligonucleotides (RASONs) provide a direct means withwhich to attenuate such discordant gene expression and epigenetically modify disease. The attractiveness of RASONs as a new class of respiratory therapeutics derives from the fact that they can modulate disease at a point more proximal to its cause-the mRNA that will continue the supply of the disease-causing protein-than traditional drugs which target the proteins already participating in the disease. Because they target mRNA, which offers target properties vastly different from proteins, some RASONs can have dramatically longer durations of effect than their traditional pharmaceutical counterparts. Additionally, RASONs can be engineered such that they are virtually entirely metabolized within the lung to a bioinactive form, reducing or eliminating the possibility of the systemic side effects that have been associated with most, if not all, traditional respiratory pharmaceuticals. In general, RASONs can be formulated as liquids or powders, and are thus amenable to delivery by any of the common devices available for respiratory drugs, including nebulizers, metered-dose inhalers, and dry powder inhalers. They are stable for long periods at room temperature, and their synthesis and purification are straightforward. Potentially long durations of effects, and the apparent assistance of pulmonary surfactant in RASON uptake and distribution throughout the lung, combine to improve the pharmacoeconomic characteristics sufficiently to be cost competitive with most currently availablerespiratory medications.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 04/04/20 alle ore 12:27:36