Lipid Nanoparticle Delivery Systems to Enable mRNA-Based Therapeutics.
Lipid Nanoparticle Delivery Systems to Enable mRNA-Based Therapeutics.
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DOI：
10.3390/pharmaceutics14020398
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发表时间：
2022-02-11
期刊：
影响因子：
5.4
通讯作者：
Lin PJC
中科院分区：
文献类型：
--
作者：
Semple SC;Leone R;Barbosa CJ;Tam YK;Lin PJC
在新冠肺炎被确认为全球大流行后，全世界都在竞相开发疫苗，以防止新冠病毒感染的迅速传播。对广泛的候选疫苗进行了评估，以mrna为基础的疫苗因其在提供高水平功效的同时可迅速用于紧急用途而成为领先者。作为一种模块化技术，基于mRNA的疫苗受益于当前全球大流行之前mRNA和递送技术几十年的进步。mRNA作为一种治疗手段的基本经验是由Katalin Kariko博士和她的同事们首创的，也许最值得注意的是与宾夕法尼亚大学的Drew Weissman合作，这项基础工作为开发有史以来第一个授权用于人类的基于mRNA的治疗药物COMIRNATY®铺平了道路。在本期《药剂学》特刊中，我们将向Kariko博士致敬，因为她在mRNA技术治疗未满足需求的疾病方面做出了巨大贡献。在这篇综述文章中，我们将重点关注传递平台，脂质纳米颗粒（LNP）载体，这使得mRNA治疗的潜力得以实现。与mRNA技术类似，LNP系统的开发已经持续了几十年，直到第一个临床批准的siRNA-LNP产品ONPATTRO®的成功，该产品用于治疗一种致命的遗传性疾病，称为转甲状腺蛋白淀粉样变性。从siRNA-LNP经验中吸取的经验教训使mRNA平台得以翻译，并最终授权和批准了针对COVID-19的mRNA- lnp疫苗。这标志着mRNA-LNP作为一种治疗遗传疾病的药物选择的开始。
The world raced to develop vaccines to protect against the rapid spread of SARS-CoV-2 infection upon the recognition of COVID-19 as a global pandemic. A broad spectrum of candidates was evaluated, with mRNA-based vaccines emerging as leaders due to how quickly they were available for emergency use while providing a high level of efficacy. As a modular technology, the mRNA-based vaccines benefitted from decades of advancements in both mRNA and delivery technology prior to the current global pandemic. The fundamental lessons of the utility of mRNA as a therapeutic were pioneered by Dr. Katalin Kariko and her colleagues, perhaps most notably in collaboration with Drew Weissman at University of Pennsylvania, and this foundational work paved the way for the development of the first ever mRNA-based therapeutic authorized for human use, COMIRNATY®. In this Special Issue of Pharmaceutics, we will be honoring Dr. Kariko for her great contributions to the mRNA technology to treat diseases with unmet needs. In this review article, we will focus on the delivery platform, the lipid nanoparticle (LNP) carrier, which allowed the potential of mRNA therapeutics to be realized. Similar to the mRNA technology, the development of LNP systems has been ongoing for decades before culminating in the success of the first clinically approved siRNA-LNP product, ONPATTRO®, a treatment for an otherwise fatal genetic disease called transthyretin amyloidosis. Lessons learned from the siRNA-LNP experience enabled the translation into the mRNA platform with the eventual authorization and approval of the mRNA-LNP vaccines against COVID-19. This marks the beginning of mRNA-LNP as a pharmaceutical option to treat genetic diseases.
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影响因子：
5.4
作者：
Bulbake U;Doppalapudi S;Kommineni N;Khan W
通讯作者：
Khan W
DOI：
10.1073/pnas.1306529110
发表时间：
2013-08-06
影响因子：
11.1
作者：
Alabi, Christopher A.;Love, Kevin T.;Anderson, Daniel G.
通讯作者：
Anderson, Daniel G.
DOI：
10.1038/mtna.2012.28
发表时间：
2012-08-14
期刊：
Molecular therapy. Nucleic acids
影响因子：
--
作者：
通讯作者：
--
DOI：
10.1056/nejmoa2035389
发表时间：
2021-02-04
期刊：
The New England journal of medicine
影响因子：
--
作者：
Baden LR;El Sahly HM;Essink B;Kotloff K;Frey S;Novak R;Diemert D;Spector SA;Rouphael N;Creech CB;McGettigan J;Khetan S;Segall N;Solis J;Brosz A;Fierro C;Schwartz H;Neuzil K;Corey L;Gilbert P;Janes H;Follmann D;Marovich M;Mascola J;Polakowski L;Ledgerwood J;Graham BS;Bennett H;Pajon R;Knightly C;Leav B;Deng W;Zhou H;Han S;Ivarsson M;Miller J;Zaks T;COVE Study Group
通讯作者：
COVE Study Group
影响因子：
5.5
作者：
Aldrich C;Leroux-Roels I;Huang KB;Bica MA;Loeliger E;Schoenborn-Kellenberger O;Walz L;Leroux-Roels G;von Sonnenburg F;Oostvogels L
通讯作者：
Oostvogels L