Review
Chemistry, Multidisciplinary
Weiqi Cai, Tianli Luo, Lanqun Mao, Ming Wang
Summary: Recent innovations in genome editing have led to precise manipulation of genetic information in mammals using stimuli-responsive nanoparticles for the delivery and release of CRISPR/Cas9 genome editing machinery. These approaches can utilize biological signals or external signals to achieve improved genome editing outcomes.
ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
(2021)
Review
Medicine, Research & Experimental
Pardis Kazemian, Si-Yue Yu, Sarah B. Thomson, Alexandra Birkenshaw, Blair R. Leavitt, Colin J. D. Ross
Summary: Gene editing mediated by CRISPR/Cas9 systems is a promising therapeutic option for genetic diseases and cancers. Lipid nanoparticles (LNPs), due to their low immunogenicity and flexibility, have emerged as an attractive nonviral delivery platform for CRISPR-mediated genome editing. The development of new, safe, and optimized LNP formulations has addressed the challenges in CRISPR delivery.
MOLECULAR PHARMACEUTICS
(2022)
Review
Medicine, Research & Experimental
Asma Ghaemi, Elnaz Bagheri, Khalil Abnous, Seyed Mohammad Taghdisi, Mohammad Ramezani, Mona Alibolandi
Summary: The prokaryotic CRISPR-Cas systems serve as a revolutionary genome editing tool for modifying, visualizing, and identifying DNA and RNA sequences in live cells of various species. Besides genome editing, the DNA-targeting modifications by CRISPR-Cas offer opportunities for diagnosis, therapy, and genetic disorders investigation. Preclinical studies and CRISPR-Cas9-based therapeutics may encounter challenges, but there is potential for its development in cancer therapy.
Review
Chemistry, Multidisciplinary
Ke Yi, Huimin Kong, Yeh-Hsing Lao, Di Li, Rachel L. Mintz, Tianxu Fang, Guojun Chen, Yu Tao, Mingqiang Li, Jianxun Ding
Summary: CRISPR/Cas9 gene-editing technology has great potential in cancer treatment, but limitations of viral delivery have led to the development of non-viral nanomaterials. Optimizing the packaging capacity, pharmacokinetics, and targetability of non-viral CRISPR/Cas9 nanoformulations can enhance the safety and efficacy of cancer therapy.
ADVANCED MATERIALS
(2023)
Review
Chemistry, Multidisciplinary
Deepak Kumar Sahel, Lalitkumar K. Vora, Aishwarya Saraswat, Saurabh Sharma, Jasmin Monpara, Anisha A. D'Souza, Deepakkumar Mishra, Kamatham Pushpa Tryphena, Satoru Kawakita, Shahid Khan, Mohd Azhar, Dharmendra Kumar Khatri, Ketan Patel, Raghu Raj Singh Thakur
Summary: CRISPR and Cas9, initially discovered as an immune system in bacteria and archaea, are now widely used as biotechnological tools for genetic and nongenetic disease treatment. Clinical trials mainly adopt ex vivo strategies, but in vivo delivery methods for CRISPR components are still under preclinical surveillance.
Review
Pharmacology & Pharmacy
Hanan Bloomer, Jennifer Khirallah, Yamin Li, Qiaobing Xu
Summary: The CRISPR/Cas system has revolutionized the ability to edit the mammalian genome, providing a platform for correcting pathogenic mutations and studying gene function. Cas9 RNPs, as a delivery method, offer advantages in editing efficiency, off-target editing activity, and cellular toxicity reduction.
ADVANCED DRUG DELIVERY REVIEWS
(2022)
Article
Chemistry, Physical
Juhee Lee, Yoo Kyung Kang, Eonju Oh, Juhee Jeong, San Hae Im, Duk Ki Kim, Haeshin Lee, Sang-Gyu Kim, Keehoon Jung, Hyun Jung Chung
Summary: The study presents a cancer gene therapy strategy based on NanoRNP that efficiently blocks the PD-L1 immune checkpoint and induces an antitumor effect in vivo without the need for combination therapy. In vivo results demonstrate that NanoRNP can induce indels in target cells at high frequencies, significantly suppressing tumor growth.
CHEMISTRY OF MATERIALS
(2022)
Review
Pharmacology & Pharmacy
Ning Ding, Sangsin Lee, Matan Lieber-Kotz, Jie Yang, Xue Gao
Summary: With over 466 million people worldwide affected by hearing loss, genetic factors contributing to 50% of congenital hearing loss, CRISPR-Cas9 systems offer new gene therapy strategies for treating genetic hearing impairment. This technology shows promise in targeted gene editing for deafness-associated genes and has the potential to revolutionize the treatment of genetic hearing diseases.
ADVANCED DRUG DELIVERY REVIEWS
(2021)
Review
Chemistry, Multidisciplinary
Yinying Pu, Wencheng Wu, Huijing Xiang, Yu Chen, Huixiong Xu
Summary: The CRISPR/Cas9 gene editing technology has the potential to cure cancer by precisely manipulating cancer-related targets. By combining gene therapy with other therapeutic modalities, this approach can achieve optimal curative effects against cancer and lay the foundation for the development of CRISPR/Cas9-based synergistic cancer therapy.
Review
Genetics & Heredity
Liangliang Ma, Shanglun Yang, Qianya Peng, Jingping Zhang, Jing Zhang
Summary: Sickle cell disease (SCD) is a common monogenic hematologic disorder caused by a point mutation in the beta-globin gene on chromosome 11. Treatment options for SCD have been limited, but gene therapy using CRISPR/Cas9 has shown promise. This paper reviews the pathogenesis and therapeutic approaches of SCD, summarizes the delivery strategies of CRISPR/Cas9, and discusses the current status and challenges of using CRISPR/Cas9 in SCD.
Review
Pharmacology & Pharmacy
Mujeeb Ur Rahman, Muhammad Bilal, Junaid Ali Shah, Ajeet Kaushik, Pierre-Louis Teissedre, Malgorzata Kujawska
Summary: Parkinson's disease and other neurodegenerative diseases have a significant impact on individuals and society. Recent research has identified neuroinflammation, oxidative stress, dysregulated protein degradation, and mitochondrial dysfunction as primary causes of Parkinson's disease. Gene-editing technologies hold promise for new treatment options.
Article
Multidisciplinary Sciences
Yeh-Hsing Lao, Robin Ji, Joyce K. Zhou, Kathy J. Snow, Nancy Kwon, Ethan Saville, Siyu He, Shradha Chauhan, Chun-Wei Chi, Malika S. Datta, Hairong Zhang, Chai Hoon Quek, S. Sarah Cai, Mingqiang Li, Yaned Gaitan, Lawrence Bechtel, Shih-Ying Wu, Cathleen M. Lutz, Raju Tomer, Stephen A. Murray, Alejandro Chavez, Elisa E. Konofagou, Kam W. Leong
Summary: This study demonstrates a safe and effective gene editing technique by using focused ultrasound to transiently open the blood-brain barrier for the transport of CRISPR/Cas9 machinery to the brain.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
(2023)
Article
Chemistry, Multidisciplinary
Honglin Tang, Xiaojie Xu, Yuxuan Chen, Huhu Xin, Tao Wan, Bowen Li, Hongming Pan, Da Li, Yuan Ping
Summary: The photothermal genome-editing strategy using a supramolecular cationic gold nanorod for CRISPR/Cas9 delivery to disrupt PD-L1 and induce immunogenic cell death (ICD) shows promising results in improving immune checkpoint blockade (ICB) therapy. This approach enhances dendritic cell conversion to T cells, promotes infiltration of cytotoxic T lymphocytes into tumors, and reprograms the immunosuppressive tumor microenvironment into an immunoactive one to inhibit primary and metastatic tumor activity with long-term immune memory effects.
ADVANCED MATERIALS
(2021)
Review
Biochemistry & Molecular Biology
Chao Zhang, Shanhe Liu, Xuan Li, Ruixuan Zhang, Jun Li
Summary: CRISPR/Cas-based genome editing technologies have revolutionized plant science by allowing precise manipulation of plant genomes. Virus-induced genome editing (VIGE) has emerged as a powerful method for delivering CRISPR/Cas reagents into plant cells, with high editing efficiency and simplified process. This article provides an overview of CRISPR/Cas-based genome editing and focuses on the VIGE systems, types of viruses used for CRISPR/Cas9 cassette delivery, recent applications, and challenges and potential of VIGE in plants.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Chemistry, Multidisciplinary
Ruosen Xie, Xiuxiu Wang, Yuyuan Wang, Mingzhou Ye, Yi Zhao, Brian S. Yandell, Shaoqin Gong
Summary: This study reports the development of a facilely fabricated pH-responsive polymer nanoparticle for safe and efficient delivery of CRISPR-associated protein 9 (Cas9) for gene editing. These nanoparticles possess many desirable properties, making them potential candidates for clinical applications in genome editing.
ADVANCED MATERIALS
(2022)
