Article
Multidisciplinary Sciences
Ni Cheng, Yaping Zhang, M. Keegan Delaney, Can Wang, Yanyan Bai, Randal A. Skidgel, Xiaoping Du
Summary: The study demonstrates that targeting integrin outside-in signaling in leukocytes and platelets can alleviate inflammation and clotting in septic mice, improving survival rates.
NATURE COMMUNICATIONS
(2021)
Article
Cell Biology
Carlos Palma-Flores, Luis Javier Cano-Martinez, Francisca Fernandez-Valverde, Itzel Torres-Perez, Sergio de los Santos, J. Manuel Hernandez-Hernandez, Adriana Fabiola Hernandez-Herrera, Silvia Garcia, Patricia Canto, Alejandro Zentella-Dehesa, Ramon Mauricio Coral-Vazquez
Summary: The study aimed to explore the expression of proteins involved in the repair process in different muscles at an early stage of muscular dystrophy. The results showed that muscles with different metabolic characteristics showed distinct expression patterns of proteins, which could be relevant in designing therapies for genetic and acquired myopathy.
JOURNAL OF MOLECULAR HISTOLOGY
(2023)
Article
Immunology
Jiali Ni, Xiaoying Li, Xiaodi Tu, Haiyan Zhu, Shiqi Wang, Yayi Hou, Huan Dou
Summary: This study found that Halofuginone (HF), a traditional Chinese medicine, could alleviate the progression of lupus mice by targeting and promoting the apoptosis of myeloid-derived suppressor cells (MDSCs) through B lymphoid tyrosine kinase (Blk). This indicates the potential of HF as an immunotherapeutic agent for the treatment of systemic lupus erythematosus (SLE).
INTERNATIONAL IMMUNOPHARMACOLOGY
(2023)
Article
Biotechnology & Applied Microbiology
Haiwen Li, Peipei Wang, Ethan Hsu, Kelsey M. Pinckard, Kristin I. Stanford, Renzhi Han
Summary: Limb-girdle muscular dystrophy type R25 (LGMDR25) is caused by recessive mutations in BVES encoding a cAMP-binding protein. There is currently no therapeutic treatment for LGMDR25. This study demonstrated the efficacy and safety of AAV9.BVES gene therapy in BVES-knockout mice, improving muscle function and cardiac conduction abnormalities.
Article
Engineering, Biomedical
Shuming Sun, Han Liu, Yan Hu, Yanpeng Wang, Mingri Zhao, Yijun Yuan, Yafei Han, Yingying Jing, Jin Cui, Xiaoxiang Ren, Xiao Chen, Jiacan Su
Summary: In this study, human skeletal muscle-specific ssDNA aptamers were selected and identified using SELEX. The aptamers showed preferential interaction with human skeletal muscle cells in vitro and specifically targeted these cells in vivo. The formation of a disulfide bond with nanoliposomes did not affect the ability of the aptamers to target skeletal muscle cells. This newly identified ssDNA aptamer and nanoliposome modification have potential applications in the treatment of human skeletal muscle diseases.
BIOACTIVE MATERIALS
(2023)
Article
Immunology
Shunli Tang, Changyi Yang, Sheng Li, Yuwei Ding, Dingxian Zhu, Shuni Ying, Chuanyin Sun, Yu Shi, Jianjun Qiao, Hong Fang
Summary: The study suggests that GSDMD may serve as a marker for elevated MAS symptoms in patients with adult-onset Still's disease, and genetic deletion or pharmacological inhibition of GSDMD can alleviate MAS symptoms and reduce IL-18 production in mice.
JOURNAL OF AUTOIMMUNITY
(2022)
Article
Physiology
Xia Dong, Tiankun Hui, Jie Chen, Zheng Yu, Dongyan Ren, Suqi Zou, Shunqi Wang, Erkang Fei, Huifeng Jiao, Xinsheng Lai
Summary: Metformin treatment improves muscle function and diminishes neuromuscular deficits in mdx mice, suggesting its potential use as a therapeutic drug in DMD patients.
FRONTIERS IN PHYSIOLOGY
(2021)
Article
Multidisciplinary Sciences
Sung-Il Yun, Sang-Kyu Lee, Eun-Ah Goh, Oh Seung Kwon, Woorim Choi, Jangseon Kim, Mi Sun Lee, Soon Ja Choi, Seung Sik Lim, Tae Kee Moon, Sin Hae Kim, Keeyeol Kyong, Gaewon Nam, Han-Oh Park
Summary: This study found that selectively inhibiting the expression of androgen receptor (AR) using self-assembled micelle inhibitory RNA (SAMiRNA) nanoparticle-type siRNA can effectively treat androgenetic alopecia (AGA). Clinical studies confirmed that this treatment increases hair counts without side effects.
SCIENTIFIC REPORTS
(2022)
Article
Gastroenterology & Hepatology
Ke-Xin Zheng, Shou-Li Yuan, Meng Dong, Han-Lin Zhang, Xiao-Xiao Jiang, Chun-Long Yan, Rong-Cai Ye, Hui-Qiao Zhou, Li Chen, Rui Jiang, Zi-Yu Cheng, Zhi Zhang, Qi Wang, Wan-Zhu Jin, Wen Xie
Summary: In this study, we identified dihydroergotamine (DHE) as a potential drug for treating liver fibrosis by targeting TGF beta R2 and inhibiting the TGF beta signaling pathway. DHE was shown to reduce fibrotic gene expressions and improve liver fibrosis in both cellular and mouse models.
WORLD JOURNAL OF GASTROENTEROLOGY
(2023)
Article
Materials Science, Multidisciplinary
Youyun Zeng, Yiding Shen, Shuyi Wu, Lei Cai, Zhen Wang, Kexin Cai, Jiating Shen, Kendrick Hii Ru Yie, Hualin Zhang, Lihua Xu, Jinsong Liu
Summary: In this study, a bone targeting drug delivery system was developed to enhance the effectiveness of anti-osteoporotic drugs, showing potential for the treatment of osteoporosis.
MATERIALS & DESIGN
(2022)
Article
Medicine, Research & Experimental
Linglong Xiao, Huaping Zheng, Jing Li, Meiqin Zeng, Dian He, Jianhao Liang, Kaijian Sun, Yunhao Luo, Feng Li, Baohong Ping, Wen Yuan, Hongwei Zhou, Qinghua Wang, Haitao Sun
Summary: This study investigated the effects of selective NLRP3 inflammasome inhibitor MCC950 on gut microbiota and corticospinal tract (CST) injury after intracerebral hemorrhage (ICH). The results showed that MCC950 modulated gut microbiota, alleviated CST injury located in the cervical enlargement of the spinal cord, and ameliorated neurological deficits after ICH.
BIOMEDICINE & PHARMACOTHERAPY
(2022)
Article
Immunology
Xiaofang Li, Pengfang Zheng, Wa Cao, Yang Cao, Xiaojun She, Honglian Yang, Kefeng Ma, Fangshan Wu, Xiujie Gao, Yu Fu, Jiayi Yin, Fei Wei, Shoufang Jiang, Bo Cui
Summary: This study investigated the effect of Lactobacillus rhamnosus GG (LGG) intervention on noise-induced cognitive deficits and systemic inflammation in rats. The results showed that LGG intervention improved noise-induced memory deterioration, promoted the proliferation of beneficial bacteria, inhibited the growth of harmful bacteria, improved dysregulation of short-chain fatty acid-producing bacteria, and regulated short-chain fatty acid levels. Mechanistically, LGG intervention alleviated the decrease in tight junction proteins in the gut and hippocampus, as well as the increase in serum inflammatory mediators induced by noise exposure. In conclusion, LGG intervention protected against cognitive deficits and systemic inflammation in rats exposed to chronic noise by reducing gut bacterial translocation, restoring gut and blood-brain barrier functions, and improving gut bacterial balance through modulation of the gut-brain axis.
FRONTIERS IN CELLULAR AND INFECTION MICROBIOLOGY
(2023)
Article
Medicine, Research & Experimental
Young-Eun Seo, Stephen H. Baine, Amber N. Kempton, Oliver C. Rogers, Sarah Lewis, Kaitlin Adegboye, Alex Haile, Ellyn L. Peterson, Eric R. Pozsgai, Rachael A. Potter, Louise R. Rodino-Klapac
Summary: This study successfully carried out AAV-mediated SGCG gene transfer experiments in SGCG-/- mice and observed improvements in muscle histopathology and functionality, providing insights for determining the minimal effective dose of SRP-9005 gene transfer therapy in LGMD 2C/R5 patients.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
(2023)
Article
Pharmacology & Pharmacy
Lu Liu, Xian Chen, Yifang Jiang, Yun Yuan, Luyao Yang, Qiongying Hu, Jianyuan Tang, Xianli Meng, Chunguang Xie, Xiaofei Shen
Summary: In this study, the researchers found that Brevilin A can inhibit lung injury and has anti-inflammatory activity. Brevilin A achieves the effect of improving acute lung injury by inhibiting the production of inflammatory factors and the activation of the NF-kappa B signaling pathway.
FRONTIERS IN PHARMACOLOGY
(2022)
Article
Cell Biology
Jin Zhang, Jin Zheng, Haitao Chen, Xinrong Li, Chenxiao Ye, Fan Zhang, Zewei Zhang, Qinghua Yao, Yong Guo
Summary: Curcumin has therapeutic effects on breast cancer cachexia by inhibiting tumor growth, reducing weight loss and muscle mass decline, and improving muscle function. It also decreases muscle inflammation, ubiquitination levels, and NF-KB pathway activation by regulating energy metabolism pathways.
MEDIATORS OF INFLAMMATION
(2022)
Article
Biotechnology & Applied Microbiology
Rachael A. Potter, Danielle A. Griffin, Patricia C. Sondergaard, Ryan W. Johnson, Eric R. Pozsgai, Kristin N. Heller, Ellyn L. Peterson, Kimmo K. Lehtimaki, Hillarie P. Windish, Plavi J. Mittal, Douglas E. Albrecht, Jerry R. Mendell, Louise R. Rodino-Klapac
HUMAN GENE THERAPY
(2018)
Article
Clinical Neurology
Jay S. Charleston, Frederick J. Schnell, Johannes Dworzak, Cas Donoghue, Sarah Lewis, Lei Chen, G. David Young, Anthony J. Milici, Jon Voss, Uditha DeAlwis, Bruce Wentworth, Louise R. Rodino-Klapac, Zarife Sahenk, Diane Frank, Jerry R. Mendell
Article
Medicine, Research & Experimental
Rui Xu, Ying Jia, Deborah A. Zygmunt, Megan L. Cramer, Kelly E. Crowe, Guohong Shao, Agatha E. Maki, Haley N. Guggenheim, Benjamin C. Hood, Danielle A. Griffin, Ellyn Peterson, Brad Bolon, John P. Cheatham, Sharon L. Cheatham, Kevin M. Flanigan, Louise R. Rodino-Klapac, Louis G. Chicoine, Paul T. Martin
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
(2018)
Article
Medicine, Research & Experimental
Lindsay M. Wallace, Nizar Y. Saad, Nettie K. Pyne, Allison M. Fowler, Jocelyn O. Eidahl, Jacqueline S. Domire, Danielle A. Griffin, Adam C. Herman, Zarife Sahenk, Louise R. Rodino-Klapac, Scott Q. Harper
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
(2018)
Article
Biotechnology & Applied Microbiology
Jerry R. Mendell, Louis G. Chicoine, Samiah A. Al-Zaidy, Zarife Sahenk, Kelly Lehman, Linda Lowes, Natalie Miller, Lindsay Alfano, Beverly Galliers, Sarah Lewis, Darren Murrey, Ellyn Peterson, Danielle A. Griffin, Kathleen Church, Sharon Cheatham, John Cheatham, Mark J. Hogan, Louise R. Rodino-Klapac
HUMAN GENE THERAPY
(2019)
Article
Medicine, General & Internal
Lindsay N. Alfano, Jay S. Charleston, Anne M. Connolly, Linda Cripe, Cas Donoghue, Robert Dracker, Johannes Dworzak, Helen Eliopoulos, Diane E. Frank, Sarah Lewis, Karin Lucas, Jessie Lynch, A. J. Milici, Amy Flynt, Emily Naughton, Louise R. Rodino-Klapac, Zarife Sahenk, Frederick J. Schnell, G. David Young, Jerry R. Mendell, Linda P. Lowes
Review
Biotechnology & Applied Microbiology
Damon R. Asher, Khampaseuth Thapa, Sachi D. Dharia, Navid Khan, Rachael A. Potter, Louise R. Rodino-Klapac, Jerry R. Mendell
EXPERT OPINION ON BIOLOGICAL THERAPY
(2020)
Article
Clinical Neurology
Jerry R. Mendell, Zarife Sahenk, Kelly Lehman, Carrie Nease, Linda P. Lowes, Natalie F. Miller, Megan A. Iammarino, Lindsay N. Alfano, Amanda Nicholl, Samiah Al-Zaidy, Sarah Lewis, Kathleen Church, Richard Shell, Linda H. Cripe, Rachael A. Potter, Danielle A. Griffin, Eric Pozsgai, Ashish Dugar, Mark Hogan, Louise R. Rodino-Klapac
Article
Biotechnology & Applied Microbiology
Danielle A. Griffin, Eric R. Pozsgai, Kristin N. Heller, Rachael A. Potter, Ellyn L. Peterson, Louise R. Rodino-Klapac
Summary: The study tested the therapeutic efficacy of a self-complementary AAVrh74 vector in sgca(-/-) mice modeling LGMD2D/R3, showing successful improvement in muscle function, histopathology, and locomotor activity. The gene therapy approach was deemed safe and effective for potential clinical application.
HUMAN GENE THERAPY
(2021)
Article
Biotechnology & Applied Microbiology
Rachael A. Potter, Danielle A. Griffin, Kristin N. Heller, Ellyn L. Peterson, Emma K. Clark, Jerry R. Mendell, Louise R. Rodino-Klapac
Summary: Duchenne muscular dystrophy (DMD) is a rare, X-linked, fatal neuromuscular disease caused by mutations in the DMD gene, with interest in gene transfer therapy for treatment. A study showed that systemic delivery of a minidystrophin gene using an adeno-associated virus vector can potentially increase muscle strength and improve muscle tissue condition in DMD patients.
HUMAN GENE THERAPY
(2021)
Review
Biotechnology & Applied Microbiology
Jerry R. Mendell, Samiah A. Al-Zaidy, Louise R. Rodino-Klapac, Kimberly Goodspeed, Steven J. Gray, Christine N. Kay, Sanford L. Boye, Shannon E. Boye, Lindsey A. George, Stephanie Salabarria, Manuela Corti, Barry J. Byrne, Jacques P. Tremblay
Summary: Hereditary diseases are caused by gene mutations, affecting millions of Americans, and gene therapy using adeno-associated virus (AAV) has shown promise in treating these diseases. Five treatments have been approved for commercialization, with many more in clinical trials, showcasing the potential of gene therapy in treating a wide range of genetic disorders.
Letter
Medicine, General & Internal
Rebecca J. Willcocks, Sean C. Forbes, Glenn A. Walter, Lee Sweeney, Louise R. Rodino-Klapac, Jerry R. Mendell, Krista Vandenborne
Summary: This case-control study investigates the impact of treatment with recombinant adeno-associated virus serotype rh74 (rAAVrh74) on muscle quality in children with Duchenne muscular dystrophy using magnetic resonance imaging and spectroscopy techniques.
Article
Clinical Neurology
Grace Cooper-Olson, Louise R. Rodino-Klapac, Rachael A. Potter
Summary: The study found that recombinant micro-dystrophin containing the complete R1-R3 region had the highest lipid-binding affinity, suggesting it could be beneficial for treating DMD.
JOURNAL OF NEUROMUSCULAR DISEASES
(2021)
Article
Medicine, Research & Experimental
Carlee R. Giesige, Lindsay M. Wallace, Kristin N. Heller, Jocelyn O. Eidahl, Nizar Y. Saad, Allison M. Fowler, Nettie K. Pyne, Mustafa Al-Kharsan, Afrooz Rashnonejad, Gholamhossein Amini Chermahini, Jacqueline S. Domire, Diana Mukweyi, Sara E. Garwick-Coppens, Susan M. Guckes, K. John McLaughlin, Kathrin Meyer, Louise R. Rodino-Klapac, Scott Q. Harper
Article
Medicine, Research & Experimental
Kristin N. Heller, Joshua T. Mendell, Jerry R. Mendell, Louise R. Rodino-Klapac