Shu Chien-Gene Lay Department of Bioengineering, University of California, San Diego, La Jolla, CA, 92093, USA.
Adv Mater. 2024 Aug;36(31):e2301738. doi: 10.1002/adma.202301738. Epub 2024 Jun 2.
Traumatic brain injury (TBI) is a critical public health concern, yet there are no therapeutics available to improve long-term outcomes. Drug delivery to TBI remains a challenge due to the blood-brain barrier and increased intracranial pressure. In this work, a chemical targeting approach to improve delivery of materials to the injured brain, is developed. It is hypothesized that the provisional fibrin matrix can be harnessed as an injury-specific scaffold that can be targeted by materials via click chemistry. To accomplish this, the brain clot is engineered in situ by delivering fibrinogen modified with strained cyclooctyne (SCO) moieties, which incorporated into the injury lesion and is retained there for days. Improved intra-injury capture and retention of diverse, clickable azide-materials including a small molecule azide-dye, 40 kDa azide-PEG nanomaterial, and a therapeutic azide-protein in multiple dosing regimens is subsequently observed. To demonstrate therapeutic translation of this approach, a reduction in reactive oxygen species levels in the injured brain after delivery of the antioxidant catalase, is achieved. Further, colocalization between azide and SCO-fibrinogen is specific to the brain over off-target organs. Taken together, a chemical targeting strategy leveraging endogenous clot formation is established which can be applied to improve therapeutic delivery after TBI.
创伤性脑损伤(TBI)是一个严重的公共卫生问题,但目前尚无改善长期预后的治疗方法。由于血脑屏障和颅内压升高,药物输送到 TBI 仍然是一个挑战。在这项工作中,开发了一种化学靶向方法来改善材料向受伤大脑的输送。假设临时纤维蛋白基质可以被利用为损伤特异性支架,通过点击化学可以靶向材料。为了实现这一目标,通过递送带有应变环辛炔(SCO)部分的纤维蛋白原来原位工程化脑凝块,这些部分整合到损伤病变中并在那里保留数天。随后观察到多种点击化学可及的、可点击的叠氮化物材料(包括小分子叠氮染料、40 kDa 叠氮 PEG 纳米材料和治疗性叠氮蛋白)在多种给药方案中的损伤内捕获和保留得到改善。为了证明这种方法的治疗转化,在递送抗氧化剂过氧化氢酶后,受伤大脑中的活性氧水平降低。此外,叠氮化物和 SCO-纤维蛋白原之间的共定位是针对大脑的,而不是针对靶外器官的。总之,建立了一种利用内源性血栓形成的化学靶向策略,可用于改善 TBI 后的治疗药物输送。