Article
Hematology
Manjola Balliu, Laura Calabresi, Niccolo Bartalucci, Simone Romagnoli, Laura Maggi, Rossella Manfredini, Matteo Lulli, Paola Guglielmelli, Alessandro Maria Vannucchi
Summary: Research shows that CALR mutations lead to abnormal activation of the IL-6 signaling pathway, promoting JAK2/STAT5 signaling through interaction with MPL. Targeting IL-6 signaling can inhibit proliferation of CALR DEL and CALR knockout cells, demonstrating therapeutic potential for treating MPNs.
Review
Medicine, Research & Experimental
Ibtissem El Ouar, Abdelhamid Djekoun
Summary: CRISPR, a family of DNA repeats, provides immunity against viral and plasmid DNA in bacteria and archaea by cleaving DNA double strands at specific sites using endonuclease Cas guided by RNA sequence. The discovery revolutionized genetic engineering, allowing for precise genome editing. While CRISPR/Cas system has vast applications in biomedical field, ethical concerns may hinder its full potential.
BIOMEDICINE & PHARMACOTHERAPY
(2021)
Article
Multidisciplinary Sciences
Liyang Zhang, John A. Zuris, Ramya Viswanathan, Jasmine N. Edelstein, Rolf Turk, Bernice Thommandru, H. Tomas Rube, Steve E. Glenn, Michael A. Collingwood, Nicole M. Bode, Sarah F. Beaudoin, Swarali Lele, Sean N. Scott, Kevin M. Wasko, Steven Sexton, Christopher M. Borges, Mollie S. Schubert, Gavin L. Kurgan, Matthew S. McNeill, Cecilia A. Fernandez, Vic E. Myer, Richard A. Morgan, Mark A. Behlke, Christopher A. Vakulskas
Summary: AsCas12a Ultra is an engineered variant with high editing efficiency and on-target specificity, making it ideal for complex therapeutic genome editing applications in multiple cell types.
NATURE COMMUNICATIONS
(2021)
Article
Cell Biology
Hyunmin Lee, Feng Cai, Neil Kelekar, Nipun K. Velupally, Jiyeon Kim
Summary: KRAS/LKB1 co-mutant lung cancer cells exhibit an increased dependence on N-acetylglucosamine-phosphate mutase 3 (PGM3) and inhibiting PGM3 can reduce tumor growth in this aggressive subtype of NSCLC.
Review
Biochemistry & Molecular Biology
Muhammad Naeem, Mubasher Zahir Hoque, Muhammad Ovais, Chanbasha Basheer, Irshad Ahmad
Summary: The innovative CRISPR-Cas technology has enabled genetic engineers to manipulate genomes effectively, but barriers in clinical applications remain, including efficient delivery and off-target effects. Various viral and non-viral vectors are designed to address these issues, but challenges such as immune response and lack of specificity persist.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Chemistry, Multidisciplinary
Ana Paula Mulet, Magdalena Ripoll, Lorena Betancor
Summary: Biocatalysis has emerged as a promising alternative to conventional chemical processes, and CRISPR-based technologies have the potential to enhance whole-cell bacterial biotransformations for more sustainable production of industrially important products.
ACS SUSTAINABLE CHEMISTRY & ENGINEERING
(2023)
Review
Biochemistry & Molecular Biology
Tae Hyeong Kim, Seong-Wook Lee
Summary: Viral infections pose a serious threat to human health and require the development of vaccines and antiviral therapeutic agents. Genome editing technology enables precise modification of genomic sequences and has expanded its applicability in developing therapeutics for viral diseases.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Multidisciplinary Sciences
Stephanie O. Erjavec, Sahar Gelfman, Alexa R. Abdelaziz, Eunice Y. Lee, Isha Monga, Anna Alkelai, Iuliana Ionita-Laza, Lynn Petukhova, Angela M. Christiano
Summary: In this study, rare variants in the KRT82 gene were identified as contributing to Alopecia Areata, and the reduced expression of KRT82 in the skin and hair follicles of individuals with Alopecia Areata was found. This study provides insight into the mechanism of rare variant predisposition and hair shaft integrity disruption in Alopecia Areata.
NATURE COMMUNICATIONS
(2022)
Review
Pharmacology & Pharmacy
Masataka Nishiga, Lei S. Qi, Joseph C. Wu
Summary: Advancements in genome editing technology, particularly the CRISPR/Cas9 system, have revolutionized biomedical research and hold great promise for therapeutic applications in treating diseases, including those in the cardiovascular field. Therapeutic genome editing strategies are already being tested in clinical trials for hematopoietic diseases and show potential for treating a wide range of conditions.
ADVANCED DRUG DELIVERY REVIEWS
(2021)
Article
Engineering, Biomedical
Kun Huang, Daniel Zapata, Yan Tang, Yong Teng, Yamin Li
Summary: Since its discovery in 2012, CRISPRCas9 has revolutionized the field of genome engineering and has shown great potential in basic science and translational medicine. The majority of clinical trials using CRISPR-Cas9 for therapeutic applications focus on ex vivo genome editing, with only a few utilizing in vivo editing strategies. In vivo genome editing bypasses tedious procedures and can be applied to a wide range of diseases and disorders. However, the lack of safe and efficient delivery systems and concerns about off-target effects pose challenges to successful translation. This review highlights the therapeutic applications of in vivo genome editing mediated by the CRISPR-Cas9 system and discusses current challenges and opportunities in the field.
Article
Cell Biology
Jiaomeng Pan, Mao Zhang, Liangqing Dong, Shuyi Ji, Juan Zhang, Shu Zhang, Youpei Lin, Xiaoying Wang, Zhenbin Ding, Shuangjian Qiu, Daming Gao, Jian Zhou, Jia Fan, Qiang Gao
Summary: This study identifies LAPTM5 as a critical contributor to lenvatinib resistance in hepatocellular carcinoma (HCC). LAPTM5 promotes intrinsic macroautophagic/autophagic flux, leading to lenvatinib resistance. LAPTM5 expression negatively correlates with lenvatinib sensitivity in clinical HCC samples, and it could serve as a potential biomarker to predict patient response to lenvatinib.
Article
Medicine, Research & Experimental
Ritika Luthra, Simran Kaur, Kriti Bhandari
Summary: Genetic disorders and congenital abnormalities affect a significant percentage of global births and can lead to early childhood deaths. The CRISPR technology shows promise in gene editing and therapeutic applications for various diseases.
Article
Biotechnology & Applied Microbiology
Jinfu Peng, Bin Yi, Mengyao Wang, Jieqiong Tan, Zhijun Huang
Summary: This study identified four key genes (ATP1A2, MT1M, SLC6A19, and TRPV6) associated with PM2.5 toxicity, mainly in regulating the mineral absorption pathway. Downregulating these genes can alleviate cell apoptosis caused by exposure to PM2.5.
FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY
(2021)
Article
Plant Sciences
Qihui Kong, Jie Li, Shoudong Wang, Xianzhong Feng, Huixia Shou
Summary: CRISPR/Cas system, a new gene-editing technology, is widely used for genome engineering. This study provides a modified protocol for generating transgenic hairy soybean roots to evaluate the efficiency of CRISPR/Cas constructs. The results demonstrated that soybean hairy root transformation could rapidly assess the efficiency of designed gRNA sequences on genome editing, making it a useful tool for functional study and pre-screening of gRNA in CRISPR/Cas gene editing.
Article
Biochemistry & Molecular Biology
Xiaolong Cao, Yeting Zhang, Mohamed Abdulkadir, Li Deng, Thomas Fernandez, Blanca Garcia-Delgar, Julie Hagstrom, Pieter J. Hoekstra, Robert A. King, Justin Koesterich, Samuel Kuperman, Astrid Morer, Cara Nasello, Kerstin J. Plessen, Joshua K. Thackray, Lisheng Zhou, Andrea Dietrich, Jay A. Tischfield, Gary A. Heiman, Jinchuan Xing
Summary: In this study, 71 strong candidate genes associated with Tourette's Disorder were identified through high-throughput sequencing and genotyping in 13 multiplex families. These candidate genes, including both known and novel genes, were enriched in several Gene Ontology categories, providing biological insight into the etiology of TD and potential targets for future studies.
MOLECULAR PSYCHIATRY
(2021)
Article
Oncology
Anandi Krishnan, Sally Thomas
Summary: Advancements in next-generation techniques like RNA-sequencing have allowed for research into the transcriptome of anucleate blood platelets, revealing a rich platelet transcriptomic signature altered in response to disease. These platelet signatures capture information from parent cells and reflect influences of tumors, stroma, and vasculature. Despite potential for cancer diagnosis and prognosis, clinical translation of platelet transcriptomics remains limited.
BRITISH JOURNAL OF CANCER
(2022)
Article
Biotechnology & Applied Microbiology
Kira Kuesters, Ronja Saborowski, Christian Wagner, Rebecca Hamel, Jan-Dirk Spoering, Wolfgang Wiechert, Marco Oldiges
Summary: This study presents an effective method for the formation of a highly active and long-term stable BsGDH-CatIB as an immobilized enzyme for robust and convenient NADH regeneration.
MICROBIAL CELL FACTORIES
(2022)
Review
Oncology
Maria Jose Jimenez-Santos, Santiago Garcia-Martin, Coral Fustero-Torre, Tomas Di Domenico, Gonzalo Gomez-Lopez, Fatima Al-Shahrour
Summary: Tumour heterogeneity, which can be categorized into inter- or intratumour heterogeneity, is a main characteristic of cancer. Precision oncology is an emerging field that aims to design tailored treatments for each patient based on their data. This review describes the current approaches for therapy selection based on different types of tumour heterogeneity and provides bioinformatics tools for personalized treatment selection.
MOLECULAR ONCOLOGY
(2022)
Article
Biochemistry & Molecular Biology
Armi M. Chaudhari, Madhvi Joshi, Dinesh Kumar, Amrutlal Patel, Kiran Bharat Lokhande, Anandi Krishnan, Katja Hanack, Slawomir Filipek, Dorian Liepmann, Venkatesan Renugopalakrishnan, Ramasamy Paulmurugan, Chaitanya Joshi
Summary: Emerging SARS-CoV-2 variants, such as the Delta and Omicron variants, pose a persistent threat with higher transmissibility and immune escape. Computational analysis, protein modeling, molecular dynamic simulations, and experimental validation were used to explore the genomic and structural basis of these variants. The results demonstrate the potential mechanism through which the Delta and Omicron variants have evolved with higher transmissibility and immune escape compared to the wild-type strain.
COMPUTATIONAL AND STRUCTURAL BIOTECHNOLOGY JOURNAL
(2022)
Article
Oncology
Anne Marie Staehle, Jan Caspar Peeken, Georg Vladimirov, Mirjam Elisabeth Hoeness, Sarolta Bojtine Kovacs, Nikolaos Karantzelis, Albert Gruender, Christoph Koellerer, Jonas Samuel Jutzi, Heike Luise Pahl, Hans Felix Staehle
Summary: The transcription factor NFE2 is overexpressed in most patients with myeloproliferative neoplasms (MPN) and mutations in NFE2 strongly predispose for acute leukemia. Transgenic mice and mice with NFE2 mutations develop MPN and leukemia, indicating a connection between NFE2 and leukemic transformation. The histone demethylase JMJD2C is a novel target gene of NFE2 and its loss impairs the proliferation of JAK2(V617F) mutated cells, suggesting JMJD2C as a potential drug target for MPN.
Article
Hematology
Sumanth Mosale Seetharam, Yi Liu, Jason Wu, Lenn Fechter, Kanagavel Murugesan, Holden Maecker, Jason Gotlib, James Zehnder, Ramasamy Paulmurugan, Anandi Krishnan
Summary: Impaired protein homeostasis plays a significant role in myeloproliferative neoplasms (MPNs). This study identifies the downregulation of enkurin (ENKUR) and the upregulation of CDC20 as potential markers of MPN pathogenesis, suggesting a novel role for ENKUR in MPNs.
Article
Medicine, Research & Experimental
Robert A. Campbell, Bhanu Kanth Manne, Meenakshi Banerjee, Elizabeth A. Middleton, Abigail Ajanel, Hansjorg Schwertz, Frederik Denorme, Chris Stubben, Emilie Montenont, Samantha Saperstein, Lauren Page, Neal D. Tolley, Diana L. Lim, Samuel M. Brown, Colin K. Grissom, Douglas W. Sborov, Anandi Krishnan, Matthew T. Rondina
Summary: IFITM3 acts as a regulator of platelet endocytosis, hyperreactivity, and thrombosis during inflammatory stress.
JOURNAL OF CLINICAL INVESTIGATION
(2022)
Article
Pharmacology & Pharmacy
Yi Liu, Uday Kumar Sukumar, Natacha Jugniot, Sumanth Mosale Seetharam, Adith Rengaramachandran, Negar Sadeghipour, Pinaki Mukherjee, Anandi Krishnan, Tarik F. Massoud, Ramasamy Paulmurugan
Summary: A new nanosystem that can be delivered via intranasal administration has been tested in mouse models for the treatment of lung metastases in TNBC. It utilizes non-toxic gold nanoparticles coated with chitosan-beta-cyclodextrin to carry suicide genes and therapeutic miRNAs. The system predominantly accumulates in the lungs and facilitates gene therapy against TNBC lung metastases.
ADVANCED THERAPEUTICS
(2022)