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科学家杂志翻译——小型化医学
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原文链接：http://the-scientist.com/2011/10/01/opinion-miniaturizing-medicine/
重庆医科大学检验系唐湛淞初译+四川大学再生医学中心刘运洪校正。水平有限请大家指正。
Opinion: Miniaturizing Medicine
观点：小型化医学
Nanotechnology will offer doctors new ways to diagnose and treat patients, boosting efficiency and slashing costs.
纳米技术将为医生提供诊断和治疗病人的新途径，抬升效率降低成本。
By Chad Mirkin | October 1, 2011
Bill Arsenault
Nanotechnology is poised to completely transform the practice of medicine. The unique physical properties of nanomaterials hold multifaceted promise for medical applications, making nanomedicine a game-changing subfield. Researchers are developing new methods that will facilitate tracking disease markers at very early stages and monitoring them as a disease progresses. This type of tracking will bring us closer to the implementation of personalized medicine, in which we will differentiate patient populations based upon their responsiveness to candidate therapies. Recent advances in nanotechnology also offer the possibility of exquisite selectivity in the delivery of therapeutic payloads.
纳米技术将彻底改变医学现状。纳米材料的独特的物理性质保证了其在医疗上多方面应用，使纳米医学领域发生革命性改变。研究人员正在开发新的方法来促进在疾病早期过程中跟踪并监测疾病标志物，并将其作为疾病进展指标。这种监测将使我们离个性化医疗的实施更近一步，也就是我们将根据不同的患者人群的敏感性来选择治疗方法。纳米技术领域的最新进展使得高精确的靶向治疗成为可能。
These approaches have the potential to substantially lower health-care and pharmaceutical-development costs, because many expensive therapeutics are now broadly and needlessly administered, even when their effectiveness is questionable for a significant portion of the population. Furthermore, the versatility and compact size of certain nano-enabled diagnostic methods could reduce the need for massive, specialized machinery, and thus help move such tests from remote sites much closer to the site of primary patient care. Indeed, some of these nanostructure-based diagnostic systems are already FDA-cleared and available in most developed countries. In addition, a number of “nanopharmaceuticals” are in clinical trials, and a few have already gained FDA approval. Considering the innovations that are now spreading quickly throughout the field, I predict that nanomaterials will comprise a large fraction of the pharmaceuticals developed over the next decade.
这些方法有可能使保健和医药开发成本大大降低，因为现在许多疗法的昂贵是滥用造成的，甚至在其有效性被大部分人所质疑时。此外，某些纳米功能的诊断方法的通用性和紧凑的尺寸可以减少对大规模，专业化的机械的需求，从而有助于对原发性病病人的检测更近一步。事实上，这些以纳米结构为基础的诊断系统中，有些已经获得FDA的认证，并且在许多发达国家开始试用。此外，许多已经获得了FDA认证的 “纳米药”正在临床期试验中。由于目前对于纳米材料的创新在整个领域迅速蔓延，我预测，在未来十年，纳米材料将占发达国家的药品的很大一部分。
A tenet of nanotechnology is that the fastest way to develop materials with new properties is through the miniaturization of existing materials. For example, gold is a material used by jewelers and nanoscientists alike because it does not degrade or react with water or air the way iron rusts. When a chunk of gold is reduced to nanoscale particles and dispersed in solution, it loses its familiar yellow glint and turns blood red. Because the optical properties of gold particles change as they are reduced in size and because gold is nontoxic and can be custom-coated with fragments of DNA, RNA, or protein, these nanoparticles have many promising uses in biosensors and diagnostic tools, and as drug-delivery vehicles.
纳米技术的宗旨是用最快的方式开发具有新性能的材料，这可以通过对现有材料小型化来实现。例如，黄金是一种珠宝商和纳米科学家使用的材料，因为它不会被水溶解或者在空气中像铁一样生锈。当一大块黄金减小成纳米大小的粒子分解在溶液中，它便会失去我们熟悉的金黄色光芒而变成血红色。因为随着黄金粒子体积的缩小使得他的光学性质发生了变化，以及黄金的无毒性，而且它可被DNA，RNA或蛋白质片段包裹，这些纳米粒子在生物传感器和诊断工具的使用和作为药物运载工具中有着很大的前景。
Advances in nanotechnology offer the possibility of exquisite selectivity in the delivery of therapeutic payloads.
随着纳米技术的发展，为高精确的治剂输送提供了可能。
Part of nanomedicine’s allure is the possibility of faster routes to the treatment of the most pressing medical problems. Take, for example, the development of drugs that modulate gene expression; a few decades of research have shown the potential for treating and even curing some of the most debilitating diseases by targeting them at their molecular roots. We are unraveling the cellular and molecular mechanisms behind cardiovascular disease, neurological disorders like Alzheimer’s disease, and many forms of cancer. However, we have learned that conventional gene-regulation strategies based upon polymer or viral delivery approaches are not easy to implement. They suffer from poor targeting capabilities, require expensive custom-synthesized nucleic acids to stabilize the active therapeutics, and often cause significant toxicity and immunogenicity.
纳米医学吸引人的部分原因是为它能够使以更快的途径解决最紧迫的医疗问题成为可能。以调节基因表达的药物为例，几十年的研究表明，针对其分子的根源有着治疗甚至治愈一些顽固性疾病的潜力。我们正在揭示心血管疾病，神经系统疾病，如阿尔茨海默氏症，以及多种形式的癌症细胞和背后的分子机制。但是，我们了解到，在传统基因调控策略中通过聚合物或病毒输送方法是难以实施的。它们由于定位能力差，又需要通过昂贵的特异核酸合成来保证其有治疗活性的稳定，这往往会引起严重的毒性和免疫反应。
AuraSense, a company that I founded with Shad Thaxton, is developing one of the technologies my lab invented—a new class of DNA- or RNA-based therapeutics with built in delivery capabilities—into a topical gene-delivery treatment for skin diseases such as psoriasis and melanoma. Current technology requires the use of needles and excruciatingly painful micro-injections to deliver genetic material as drugs. However, by using nanoengineered gold particles with specific DNA or RNA affixed to their surfaces, the nucleic acid therapeutics of interest can be topically (and painlessly) delivered to the skin. This application allows us to target diseased cells at the genetic level, up- or downregulating pathways based upon the nucleic acids on the surface of the particles. These constructs also show promise as systemic treatments for patients with life-threatening diseases such as glioblastoma, liver cancer, and bladder cancer, to name only a few.
AuraSense，我和Shad Thaxton,一起创立的一家公司，正在研发我的实验室发明的一项技术用于局部基因输送治疗皮肤病如牛皮癣及黑色素瘤，这是一类新的基于DNA或RNA输送能力的研究。目前的技术要求使用的针头和极度痛苦的显微注射输送遗传物质的药物。然而，通过设计使用特定的贴到DNA及RNA表面的的纳米金颗粒，目的核酸可局部（无痛）传递到皮肤。这种应用使得我们在基因水平上跟踪疾病细胞，向上或向下调节颗粒表面上的核酸为基础的通路。这些设计还提示了在危及生命的疾病患者的全身治疗的看到希望，如胶质母细胞瘤，肝癌，膀胱癌等等。
On the diagnostic side, we are discovering powerful new ways of detecting and tracking disease biomarkers at very early stages—stages that cannot be detected with conventional tools. Genetic and protein-based tests developed by companies like Nanosphere, which Robert Letsinger and I cofounded, rely upon the exquisite sensitivity and selectivity of nanoparticle probes to help identify and target disease-related genetic sequences or proteins. The probes’ minute size and ease of use make it possible for a physician to perform a diagnosis concurrently with treatment, rather than sending samples to a remote lab to process via existing molecular diagnostic methods, such as ELISA or PCR-based techniques, prior to any treatment decisions. This new technology is allowing medical researchers and clinicians to diagnose heart disease or potential heart failure at the very earliest stages, when therapeutics can be more effective.
在诊断方面，我们发现了检测疾病的生物标志物检测和跟踪的强有力的新方法，能在非常早期的阶段发挥作用，而传统的方法检测不到。我和Robert Letsinger共同创立的Nanosphere公司，开发了以基因和以蛋白质为基础的测试，依靠精湛的纳米粒子探针的灵敏度和选择性来帮助识别和目标疾病相关的基因序列或蛋白质。探针的微小尺寸和易用性有可能使医生同时做到诊断与治疗，而不是在进行治疗决策之前还将样品送到遥远的实验室，通过现有的分子诊断方法，如ELISA或PCR为基础的技术进行诊断。这项新技术使医学研究人员和临床医生在最初阶段就能诊断出心脏疾病或潜在的心脏衰竭的，使治疗更加有效。
The next decade of nanomedicine will see the continued transition of therapeutic candidates from the bench top to the clinic. Examples like the FDA-approved Abraxane, for treatment of metastatic breast cancer, have shown that combining a known anticancer drug with albumin nanoparticles can have significant impact in terms of efficacy, simply by making it possible to adjust the drug’s solubility and dissolution rates. Because nanoparticles are larger than small-molecule drugs, they can carry both a targeting molecule and a therapeutic payload within one entity. They can be designed to have sizes, shapes, and internal structures that are conducive to effective delivery of therapy. Based on these ideas, companies like Liquidia and Calando are developing very promising nanotechnologies for the treatment of a vast array of diseases, including several forms of cancer, infectious diseases, and respiratory ailments.
未来十年的纳米医学将会看到治疗方案的候选由实验台到临床的持续过渡。就像FDA认证Abraxane治疗转移性乳腺癌一样，白蛋白纳米粒结合已知的抗癌药物已有显著影响疗效，通过它可以方便的调整药物的溶解度和析出率。由于纳米粒子大于小分子药物，一个纳米分子可以携带靶向分子和有效的治疗药物。他们的大小，形状，和内部结构可以被设计利于有效地提供治疗。基于这些想法，像Liquidia和Calando这样的公司正在开发繁多的非常有前途的纳米技术来治疗疾病，包括多种癌症，传染性疾病和呼吸系统疾病。
Nanostructures are also showing significant promise as “theranostic” systems, which provide the ability to image or detect disease in vivo, deliver targeted therapy triggered by the diagnostic discovery, and then monitor the response—but this technology is still being developed at the university level. These multifunctional systems rely on specific attributes of nanostructures, including their shape, surface functionality, ability to move throughout the body, and specificity. These properties are difficult, if not impossible, to realize with macroscopic and molecular approaches. As they continue to reach the clinic, nanotechnology innovations will help redefine the practice of medicine as we know it today.
纳米结构“theranostic”系统，在体内疾病的成像与探测的能力，靶向治疗诊断性发现，然后监控的其反应，但这项技术仍然是在学校水平开发。这些多功能系统依靠特定属性的纳米结构，包括它们的形状，表面的功能，整个身体的移动能力以及特异性。这些属性实现宏观和分子的方法是困难的，但这是可能的。在他们陆陆续续进入临床后，纳米技术革命将重新定义我们今天所知道的医疗。
Chad Mirkin is a professor at Northwestern University, director of the university’s International Institute for Nanotechnology, and a member of President Obama’s Council of Advisors on Science and Technology.
Chad Mirkin是在西北大学，学校国际纳米技术研究所主任的教授，奥巴马总统的科技顾问委员会的成员。
https://blog.sciencenet.cn/blog-616439-511689.html
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