A Rally for Brain Targeting: The Advent of a New Era

References

• Zlokovic BV . Neurovascular mechanisms of Alzheimer's neurodegeneration. Trends Neurosci.28, 202–208 (2005).
   PubMed Web of Science ®Google Scholar
• Attwell D , BuchanAM , CharpakSet al. Glial and neuronal control of brain blood flow. Nature468, 232–243 (2010).
   PubMed Web of Science ®Google Scholar
• Zlokovic BV . The blood-brain barrier in health and chronic neurodegenerative disorders. Neuron57, 178–201 (2008).
   PubMed Web of Science ®Google Scholar
• Dong X . Current strategies for brain drug delivery. Theranostics8, 1481–1493 (2018).
   PubMed Web of Science ®Google Scholar
• Carroll WM . The global burden of neurological disorders. Lancet Neurol.18, 418–419 (2019).
   PubMed Web of Science ®Google Scholar
• Lajoie JM , ShustaEV. Targeting receptor-mediated transport for delivery of biologics across the blood–brain barrier. Annu. Rev. Pharmacol. Toxicol.55, 613–631 (2015).
   PubMed Web of Science ®Google Scholar
• Giugliani R , GiuglianiL , deOliveira Poswar Fet al. Mucopolysaccharidosis type I after 52 weeks of intravenous brain-penetrating insulin receptor anti-iduronidase fusion protein (valanafusp alpha): an open label Phase I–II trial. Orphanet J. Rare Dis.13, 110 (2018).
   PubMed Web of Science ®Google Scholar
• van der Worp HB , HowellsDW , SenaESet al. Can animal models of disease reliably inform human studies? PLoS Med. 7, e1000245 (2010).
   PubMed Web of Science ®Google Scholar
• Bhalerao A , SivandzadeF , ArchieARet al. In vitro modelling of the neurovascular unit: advances in the field. Fluids Barriers CNS17, 22 (2020).
   PubMed Web of Science ®Google Scholar
• Sonoda H , MorimotoH , YodenEet al. A blood-brain-barrier-penetrating anti-human transferrin receptor antibody fusion protein for neuronopathic mucopolysaccharidosis II. Mol. Ther.26, 1366–1374 (2018).
   PubMed Web of Science ®Google Scholar
• Haery L , DevermanBE , MathoKSet al. Adeno-associated virus technologies and methods for targeted neuronal manipulation. Front. Neuroanat.13, 93 (2019).
   PubMed Web of Science ®Google Scholar
• Munji RN , SoungAL , WeinerGAet al. Profiling the mouse brain endothelial transcriptome in health and disease models reveals a core blood–brain barrier dysfunction module. Nat. Neurosci.22, 1892–1902 (2019).
   PubMed Web of Science ®Google Scholar
• Park IH , LerouPH , ZhaoRet al. Generation of human-induced pluripotent stem cells. Nat. Protoc.3, 1180–1186 (2008).
   PubMed Web of Science ®Google Scholar
• Bien-Ly N , YuYJ , BumbacaDet al. Transferrin receptor (TfR) trafficking determines brain uptake of TfR antibody affinity variants. J. Exp. Med.211, 233–244 (2014).
   PubMed Web of Science ®Google Scholar
• Beard R , GaboriauDCA , GeeADet al. Chemical biology tools for probing transcytosis at the blood–brain barrier. Chem. Sci.10, 10772–10778 (2019).
   PubMed Web of Science ®Google Scholar
• Villaseñor R , SchillingM , SundaresanJet al. Sorting tubules regulate blood-brain barrier transcytosis. Cell Rep.21, 3256–3270 (2017).
   PubMed Web of Science ®Google Scholar
• Moghimi SM , HowardKA. Targeting biological barriers: turning a wall into a therapeutic springboard. Mol. Ther.26, 933–934 (2018).
   PubMed Web of Science ®Google Scholar
• Clark AJ , DavisME. Increased brain uptake of targeted nanoparticles by adding an acid-cleavable linkage between transferrin and the nanoparticle core. Proc. Natl Acad. Sci. USA112, 12486–12491 (2015).
   PubMed Web of Science ®Google Scholar
• Baker GJ , YadavVN , MotschSet al. Mechanisms of glioma formation: iterative perivascular glioma growth and invasion leads to tumor progression, VEGF-independent vascularization, and resistance to antiangiogenic therapy. Neoplasia16, 543–561 (2014).
   PubMed Web of Science ®Google Scholar
• Shindhwani S , SyedAM , NgaiJet al. The entry of nanoparticles into solid tumours. Nat. Mat.19, 566–575 (2020).
   PubMed Web of Science ®Google Scholar
• Park T-E , MustafaogluN , HerlandAet al. Hypoxia-enhanced blood–brain barrier chip recapitulates human barrier function and shuttling of drugs and antibodies. Nat. Commun.10, 2621 (2019).
   PubMed Web of Science ®Google Scholar
• Ferro MP , HeilshornSC , OwensRM. Materials for blood brain barrier modelling in vitro. Mat. Sci. Eng. R140, 100522 (2020).
   PubMed Web of Science ®Google Scholar
• Cakir B , XiangY , TanakaYet al. Engineering of human brain organoids with a functional vascular-like system. Nat. Methods16, 1169–1175 (2019).
   PubMed Web of Science ®Google Scholar
• Foust KD , NurreE , MontgomeryLCet al. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat. Biotechnol.27, 59–65 (2009).
   PubMed Web of Science ®Google Scholar
• Deverman BE , PravdoPL , SimpsonBPet al. Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain. Nat. Biotechnol.34, 204–209 (2016).
   PubMed Web of Science ®Google Scholar
• Zolgensma® (onasemnogene abeparvovec-xioi) . (2020). www.zolgensma.com
   Google Scholar
• Federici T , TaubJS , BaumGRet al. Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs. Gene Ther.19, 852–859 (2012).
   PubMed Web of Science ®Google Scholar
• Schuster DJ , DykstraJA , RiedlMSet al. Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse. Front. Neuroanat.8, 42 (2014).
   PubMed Web of Science ®Google Scholar
• Kumar SR , MilesTF , ChenXet al. Multiplexed Cre-dependent selection yields systemic AAVs for targeting distinct brain cell types. Nat. Methods17, 541–550 (2020).
   PubMed Web of Science ®Google Scholar
• Albright BH , StoreyCM , MurlidharanGet al. Mapping the structural determinants required for AAVrh.10 transport across the blood-brain-barrier. Mol. Ther.26, 510–523 (2018).
   PubMed Web of Science ®Google Scholar
• Wu L-P , AhmadvandD , SuJet al. Crossing the blood-brain-barrier with nanoligand drug carriers self-assembled from a phage display peptide. Nat. Commun.10, 4635 (2019).
   PubMed Web of Science ®Google Scholar
• Mann AP , ScodellerP , HussainSet al. Identification of a peptide recognizing cerebrovascular changes in mouse models of Alzheimer's disease. Nat. Commun.8, 1403 (2017).
   PubMed Web of Science ®Google Scholar
• Mann AP , ScodellerP , HussainSet al. A peptide for targeted, systemic delivery of imaging and therapeutic compounds into acute brain injuries. Nat. Commun.7, 11980 (2016).
   PubMed Web of Science ®Google Scholar