Spotlight on Genetic Kidney Diseases: A Call for Drug Delivery and Nanomedicine Solutions

被引：22

作者：

Trac, Noah ^{[1
]}

Ashraf, Anisa ^{[1
]}

Giblin, Joshua ^{[1
]}

Prakash, Supriya ^{[2
,3
]}

Mitragotri, Samir ^{[2
,3
]}

Chung, Eun Ji ^{[1
,4
,5
,6
]}

机构：

[1] Univ Southern Calif, Dept Biomed Engn, Los Angeles, CA 90089 USA

[2] Harvard Univ, John A Paulson Sch Engn & Appl Sci, Allston, MA 02134 USA

[3] Wyss Inst Biol Inspired Engn, Boston, MA 02115 USA

[4] Univ Southern Calif, Mork Family Dept Chem Engn & Mat Sci, Los Angeles, CA 90089 USA

[5] Univ Southern Calif, Dept Med,Eli & Edythe Broad Ctr Regenerat Med & S, Keck Sch Med, Norris Comprehens Canc Ctr, Los Angeles, CA 90033 USA

[6] Univ Southern Calif, Dept Surg, Div Vasc Surg & Endovasc Therapy, Keck Sch Med, Los Angeles, CA 90033 USA

来源：

ACS NANO | 2023年 / 17卷 / 07期

关键词：

genetic kidney disease; congenital disease; nanomedicine; cell therapy; drug delivery; rare diseases; IN-VITRO SELECTION; NANOPARTICLES; PEPTIDE; CELLS; PLACENTA; THERAPY; LIGANDS; DNA; OPPORTUNITIES; STRATEGIES;

D O I：

10.1021/acsnano.2c12140

中图分类号：

O6 [化学];

学科分类号：

0703 ;

摘要：

Nanoparticles as drug delivery carriers have benefited diseases, including cancer, since the 1990s, and more recently, their promise to quickly and efficiently be mobilized to fight against global diseases such as in the COVID-19 pandemic have been proven. Despite these success stories, there are limited nanomedicine efforts for chronic kidney diseases (CKDs), which affect 844 million people worldwide and can be linked to a variety of genetic kidney diseases. In this Perspective, we provide a brief overview of the clinical status of genetic kidney diseases, background on kidney physiology and a summary of nanoparticle design that enable kidney access and targeting, and emerging technological strategies that can be applied for genetic kidney diseases, including rare and congenital kidney diseases. Finally, we conclude by discussing gaps in knowledge remaining in both genetic kidney diseases and kidney nanomedicine and collective efforts that are needed to bring together stakeholders from diverse expertise and industries to enable the development of the most relevant drug delivery strategies that can make an impact in the clinic.

引用

页码：6165 / 6177

页数：13

共 95 条

[1]

ABRAHAMSON DR, 1991, SEMIN NEPHROL, V11, P375

[2] The CRISPR tool kit for genome editing and beyond [J].

Adli, Mazhar .

NATURE COMMUNICATIONS, 2018, 9

[3] The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs [J].

Akinc, Akin ;

Maier, Martin A. ;

Manoharan, Muthiah ;

Fitzgerald, Kevin ;

Jayaraman, Muthusamy ;

Barros, Scott ;

Ansell, Steven ;

Du, Xinyao ;

Hope, Michael J. ;

Madden, Thomas D. ;

Mui, Barbara L. ;

Semple, Sean C. ;

Tam, Ying K. ;

Ciufolini, Marco ;

Witzigmann, Dominik ;

Kulkarni, Jayesh A. ;

van der Meel, Roy ;

Cullis, Pieter R. .

NATURE NANOTECHNOLOGY, 2019, 14 (12) :1084-1087

[4] A high-throughput screening platform for Polycystic Kidney Disease (PKD) drug repurposing utilizing murine and human ADPKD cells [J].

Asawa, Rosita R. ;

Danchik, Carina ;

Zahkarov, Alexey ;

Chen, Yuchi ;

Voss, Ty ;

Jadhav, Ajit ;

Wallace, Darren P. ;

Trott, Josephine F. ;

Weiss, Robert H. ;

Simeonov, Anton ;

Martinez, Natalia J. .

SCIENTIFIC REPORTS, 2020, 10 (01)

[5] Mutations in six nephrosis genes delineate a pathogenic pathway amenable to treatment [J].

Ashraf, Shazia ;

Kudo, Hiroki ;

Rao, Jia ;

Kikuchi, Atsuo ;

Widmeier, Eugen ;

Lawson, Jennifer A. ;

Tan, Weizhen ;

Hermle, Tobias ;

Warejko, Jillian K. ;

Shril, Shirlee ;

Airik, Merlin ;

Jobst-Schwan, Tilman ;

Lovric, Svjetlana ;

Braun, Daniela A. ;

Gee, Heon Yung ;

Schapiro, David ;

Majmundar, Amar J. ;

Sadowski, Carolin E. ;

Pabst, Werner L. ;

Daga, Ankana ;

van der Ven, Amelie T. ;

Schmidt, Johanna M. ;

Low, Boon Chuan ;

Gupta, Anjali Bansal ;

Tripathi, Brajendra K. ;

Wong, Jenny ;

Campbell, Kirk ;

Metcalfe, Kay ;

Schanze, Denny ;

Niihori, Tetsuya ;

Kaito, Hiroshi ;

Nozu, Kandai ;

Tsukaguchi, Hiroyasu ;

Tanaka, Ryojiro ;

Hamahira, Kiyoshi ;

Kobayashi, Yasuko ;

Takizawa, Takumi ;

Funayama, Ryo ;

Nakayama, Keiko ;

Aoki, Yoko ;

Kumagai, Naonori ;

Iijima, Kazumoto ;

Fehrenbach, Henry ;

Kari, Jameela A. ;

El Desoky, Sherif ;

Jalalah, Sawsan ;

Bogdanovic, Radovan ;

Stajic, Natasa ;

Zappel, Hildegard ;

Rakhmetova, Assel .

NATURE COMMUNICATIONS, 2018, 9

[6] Selection of peptide ligands binding to the basolateral cell surface of proximal convoluted tubules [J].

Audigé, A ;

Frick, C ;

Frey, FJ ;

Mazzucchelli, L ;

Odermatt, A .

KIDNEY INTERNATIONAL, 2002, 61 (01) :342-348

[7] Kidney transplant: New opportunities and challenges [J].

Augustine, Joshua .

CLEVELAND CLINIC JOURNAL OF MEDICINE, 2018, 85 (02) :138-144

[8] Genetic Etiologies for Chronic Kidney Disease Revealed through Next-Generation Renal Gene Panel [J].

Bleyer, Anthony J. ;

Westemeyer, Maggie ;

Xie, Jing ;

Bloom, Michelle S. ;

Brossart, Katya ;

Eckel, Jason J. ;

Jones, Frederick ;

Molnar, Miklos Z. ;

Kotzker, Wayne ;

Anand, Prince ;

Kmoch, Stanislav ;

Xue, Yuan ;

Strom, Samuel ;

Punj, Sumit ;

Demko, Zachary P. ;

Tabriziani, Hossein ;

Billings, Paul R. ;

McKanna, Trudy .

AMERICAN JOURNAL OF NEPHROLOGY, 2022, 53 (04) :297-306

[9] Formation of cysts by principal-like MDCK cells depends on the synergy of cAMP- and ATP-mediated fluid secretion [J].

Buchholz, Bjoern ;

Teschemacher, Barbara ;

Schley, Gunnar ;

Schillers, Hermann ;

Eckardt, Kai-Uwe .

JOURNAL OF MOLECULAR MEDICINE-JMM, 2011, 89 (03) :251-261

[10] Genetics of Congenital Anomalies of the Kidney and Urinary Tract: The Current State of Play [J].

Capone, Valentina P. ;

Morello, William ;

Taroni, Francesca ;

Montini, Giovanni .

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2017, 18 (04)

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