Article
Biochemistry & Molecular Biology
Huijuan Li, Hong Yu, Qi Li
Summary: The Pacific oyster's striated myosin heavy chain, CgSmhc, has high expression levels during development and is primarily expressed in muscle tissues. Various conserved binding sites of myogenesis-related regulatory elements were found in the 5'-flanking region of CgSmhc, and a 2000 bp sequence was sufficient to induce its expression. Disruption of CgSmhc using CRISPR/Cas9 had a visible effect on the sarcomeric organization of thin filaments in larval musculature, indicating its importance in regulating correct assembly during larval myogenesis.
INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES
(2021)
Article
Biochemistry & Molecular Biology
Xiaoying Zhao, Kunli Qu, Benedetta Curci, Huanming Yang, Lars Bolund, Lin Lin, Yonglun Luo
Summary: Recent progress in CRISPR gene editing tools has expanded the possibilities for treating devastating genetic diseases. In this study, three methods of gene editing (NHBEJ, HDR, and PE) were compared for correcting loss-of-function mutations in Duchenne Muscular Dystrophy. The highest efficiency was achieved with NHBEJ, followed by HDR and PE2. The correction efficiency was increased with the use of PE3. This study demonstrates the potential for highly efficient correction of DMD mutations using CRISPR gene editing.
Article
Biotechnology & Applied Microbiology
Huijuan Li, Hong Yu, Shaojun Du, Qi Li
Summary: Pacific oyster is a widely cultivated shellfish species with limited genetic tools for functional analysis. The successful application of CRISPR/Cas9 technology to knockout the CgMELC gene revealed its involvement in muscle contraction and myogenesis in oyster larvae.
MARINE BIOTECHNOLOGY
(2021)
Article
Biology
Hye In Ka, Hyemin Seo, Youngsook Choi, Joohee Kim, Mina Cho, Seok-Yong Choi, Sujeong Park, Sora Han, Jinsu An, Hak Suk Chung, Young Yang, Min Jung Kim
Summary: IK is a splicing factor that plays an essential role in embryonic development in vertebrates, promoting normal skeletal muscle development and myogenic differentiation in vivo and in vitro. Depletion of IK leads to downregulation of genes involved in skeletal muscle differentiation and improper pre-mRNA splicing, resulting in impaired neuromuscular junction and fast-twitch muscle development in zebrafish embryos.
Article
Biochemistry & Molecular Biology
Jitesh Kumar, Si Nian Char, Trevor Weiss, Hua Liu, Bo Liu, Bing Yang, Feng Zhang
Summary: Efficient and precise targeted insertion in plant genome editing remains challenging. However, a recent study combined CRISPR/Cas9 gene editing with chemically protected dsODNs and achieved high efficiency and control over the direction of targeted insertions.
Review
Biochemistry & Molecular Biology
Christopher E. Denes, Alexander J. Cole, Yagiz Alp Aksoy, Geng Li, Graham Gregory Neely, Daniel Hesselson
Summary: Genome modification holds great potential for disease prevention or treatment, with CRISPR/Cas9 techniques showing promise in altering disease-relevant genes. Competition among DNA repair pathways can lead to undesirable editing outcomes, but small molecule modulators and engineered CRISPR/Cas proteins have been shown to enhance precision editing efficacy.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
News Item
Multidisciplinary Sciences
Heidi Ledford
Summary: Base editing is being tested in CAR-T-cell treatments for leukaemia, allowing specific modifications to a cell's genome.
Review
Biochemistry & Molecular Biology
Magdalena Hryhorowicz, Daniel Lipinski, Joanna Zeyland
Summary: The bacteria-derived CRISPR/Cas system is a versatile tool for genome engineering, with wide applications in various fields. However, challenges like immunological reactions and off-target effects remain. This review discusses the current classification of CRISPR systems, new genome-editing technologies, recent applications, as well as limitations, ethical issues, and challenges associated with the CRISPR/Cas system.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Anatomy & Morphology
Minami Ezawa, Fumika Kouno, Hideo Kubo, Tetsushi Sakuma, Takashi Yamamoto, Tsutomu Kinoshita
Summary: In Xenopus development, Pou5f3.3 plays a crucial role in establishing and maintaining hematopoietic progenitor cells, contributing to the regulation of peripheral blood cell homeostasis. Transplantation experiments demonstrate the migration of Pou5f3.3-positive cells from the hematopoietic dorsal-lateral plate to the liver and bone through the bloodstream, highlighting its significance in hematopoiesis.
Article
Biochemistry & Molecular Biology
Qian Wang, Jin Liu, Josephine M. Janssen, Marie Le Bouteiller, Richard L. Frock, Manuel A. F. Goncalves
Summary: The study demonstrates that high-specificity Cas9 nucleases can be converted into nicking Cas9(D10A) variants with superior precision compared to the commonly used Cas9(D10A)nickase. Dual nicking RGNs based on these Cas9(D10A) variants can achieve gene knockouts and knock-ins at frequencies similar to or higher than conventional methods. Additionally, the high-specificity dual nicking RGNs are able to distinguish highly similar sequences and preserve genomic integrity.
NUCLEIC ACIDS RESEARCH
(2021)
Article
Cell Biology
Joe Ibarra, Tyler Hershenhouse, Luay Almassalha, David Walterhouse, Vadim Backman, Kyle L. Macquarrie
Summary: The study found chromosome-specific differences in the organizational characteristics of chromosomes during normal myogenesis, with chromosome 2 showing increased area occupation and a shift in peripheral localization. Additionally, it was discovered that the positioning of chromosome 2 changes during differentiation, with a preference for positioning along the major nuclear axis only in myotubes, whereas RMS cells do not display such preference.
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY
(2023)
Article
Cell & Tissue Engineering
Huan Zou, Mingfeng Guan, Yundong Li, Fang Luo, Wenyuan Wang, Yiren Qin
Summary: This study successfully corrected the genetic mutation of Achondroplasia patients using stem cells and gene editing technology, restoring their chondrogenic differentiation ability and providing an important theoretical and experimental basis for Achondroplasia research and treatment.
STEM CELL RESEARCH & THERAPY
(2021)
Review
Medicine, Research & Experimental
Katye M. Fichter, Tahereh Setayesh, Punam Malik
Summary: CRISPR-Cas technologies have the potential to revolutionize genetic medicine but further research is needed for clinical efficiency. The repair of CRISPR-Cas-induced DNA breaks in the human genome remains a challenge, with non-homologous end-joining leading to gene disruption. Homology-directed repair (HDR) offers precise genetic changes but requires cycling cells and a homology donor template. Newer editing strategies such as base and prime editing show promise for small edits without relying on HDR and overcoming cell cycle dependence.
MOLECULAR THERAPY-NUCLEIC ACIDS
(2023)
Review
Biotechnology & Applied Microbiology
Lam Chung Liang, Nadiah Sulaiman, Muhammad Dain Yazid
Summary: Duchenne muscular dystrophy (DMD) is a severe form of muscle dystrophy that currently lacks effective treatment options. Gene therapy has been proposed as a potential solution, but it also faces limitations and challenges.
FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY
(2022)
Article
Dermatology
Huishan Shi, Jos P. H. Smits, Ellen H. van den Bogaard, Matthew G. Brewer
Summary: The CRISPR/Cas9 technology is a powerful tool used to alter genetic landscapes in various hosts, particularly in developing novel mouse lines modeling disease states. It has been successfully used in both in vitro and in vivo systems within fields like epithelial biology and dermatology. The delivery of single-guide RNA/Cas9 editing complex to host cells is crucial for its success, and different delivery techniques have been explored and evaluated for their benefits and drawbacks in genetically modifying epidermal cells. Recent advances in dermatology have utilized these methods to better understand epidermal biology, identify therapeutic targets, and develop new treatment approaches for disease states.
JOURNAL OF INVESTIGATIVE DERMATOLOGY
(2021)
Article
Cardiac & Cardiovascular Systems
Yong Wang, Yiming Xu, Siyuan Yan, Kaixiang Cao, Xianqiu Zeng, Yaqi Zhou, Zhiping Liu, Qiuhua Yang, Yue Pan, Xiaoling Wang, Detlev Boison, Yunchao Su, Xuejun Jiang, Vijay S. Patel, David Fulton, Neal L. Weintraub, Yuqing Huo
Summary: The study reveals a novel mechanism by which ADK promotes VSMC proliferation via inducing aberrant DNA hypermethylation, down-regulating KLF4 expression and promoting neointima formation. The findings suggest the possibility of targeting ADK as an epigenetic modulator to combat vascular injury.
CARDIOVASCULAR RESEARCH
(2021)
Article
Chemistry, Multidisciplinary
Xuan Su, Yan Shen, Yue Jin, Neal L. Weintraub, Yao-liang Tang
Summary: Transplantation with allogeneic myogenic progenitor-derived exosomes (MPC-Exo) can improve heart function in X-linked muscular dystrophy (Mdx) mice by regulating various signaling pathways and physiological processes, increasing Bcl-2 expression, and decreasing IL-6 expression.
ACTA PHARMACOLOGICA SINICA
(2021)
Article
Medical Laboratory Technology
Rebekah Tritz, Tyler Benson, Valerie Harris, Farlyn Z. Hudson, James Mintz, Hanfang Zhang, Simone Kennard, Weiqin Chen, David W. Stepp, Gabor Csanyi, Eric J. Belin de Chantemele, Neal L. Weintraub, Brian K. Stansfield
Summary: Nf1+/- mice closely replicate the anthropometric and metabolic features identified in individuals with NF1, showing leaner body composition, increased insulin sensitivity, and protection from diet-induced obesity and hyperglycemia. These findings may impact the interpretation of translational studies of NF1 in the future.
TRANSLATIONAL RESEARCH
(2021)
Editorial Material
Cardiac & Cardiovascular Systems
Avirup Guha, Nihar R. Desai, Neal L. Weintraub
EUROPEAN JOURNAL OF PREVENTIVE CARDIOLOGY
(2021)
Editorial Material
Cardiac & Cardiovascular Systems
Richard Lee, Neal Weintraub
JOURNAL OF THORACIC AND CARDIOVASCULAR SURGERY
(2022)
Review
Peripheral Vascular Disease
Michael A. Winkler, Ripa Patel, Weibo Fu, Vishal Arora, Neal L. Weintraub
Summary: This review emphasizes the importance of using cardiac computed tomography, intracoronary imaging, and lesion-specific physiological guidance in guiding the decision to revascularize patients with chronic coronary syndrome.
INTERNATIONAL JOURNAL OF ANGIOLOGY
(2021)
Article
Cardiac & Cardiovascular Systems
Tatsuya Aonuma, Bruno Moukette, Satoshi Kawaguchi, Nipuni P. Barupala, Marisa N. Sepulveda, Kyle Frick, Yaoliang Tang, Maya Guglin, Subha Raman, Chenleng Cai, Suthat Liangpunsakul, Shinichi Nakagawa, Il-man Kim
Summary: In this study, the functional interaction among MIAT, miR-150, and Hoxa4 in ischemic heart failure was investigated. The findings provide valuable insights into the regulatory mechanism of these noncoding RNAs in cardiac protection.
CIRCULATION-HEART FAILURE
(2022)
Article
Cell Biology
Brandee Goo, Samah Ahmadieh, Abdalrahman Zarzour, Nicole K. H. Yiew, David Kim, Hong Shi, Jacob Greenway, Stephen Cave, Jenny Nguyen, Swetha Aribindi, Mark Wendolowski, Praneet Veerapaneni, Mourad Ogbi, Weiqin Chen, Yun Lei, Xin-Yun Lu, Ha Won Kim, Neal L. Weintraub
Summary: Obesity is a major risk factor for metabolic and cardiovascular diseases. Research has found that adipocyte-specific deletion of the HDAC9 gene can alleviate obesity and metabolic diseases in female mice, but the effect is less significant in male mice. These findings reveal sex-related differences in the contribution of HDAC9 to obesity-related metabolic diseases.
Article
Cell Biology
Yan Shen, Il-man Kim, Yaoliang Tang
Summary: This study used snRNA-seq technology to analyze the effect of DMD mutation on blood pressure and found that DMD mutation reduces blood pressure in mice and alters gene expression in VSMCs. The study also discovered that KCNQ5 gene expression is upregulated in DMDmut-VSMCs, which may be related to the mechanism of hypotension in DMD patients.
Article
Cell Biology
Haitao Zhang, Yan Shen, Il-man Kim, Yutao Liu, Jingwen Cai, Adam E. E. Berman, Kent R. R. Nilsson, Neal L. L. Weintraub, Yaoliang Tang
Summary: Clinical trials have shown that electric stimulation using cardiac resynchronization therapy or cardiac contractility modulation is effective for treating moderate to severe heart failure. This study investigated the effects of electric stimulation on the secretion of extracellular vesicles (EV) from cardiac mesenchymal stem cells (C-MSC). The results showed that electric stimulation enhanced EV-dependent cardioprotection and involved the enzyme nSMase2 in the endosomal sorting complex for EV biosynthesis.
Article
Biology
Yan Shen, Il-man Kim, Mark Hamrick, Yaoliang Tang
Summary: Duchenne muscular dystrophy (DMD) is a severe disease caused by mutations in the dystrophin gene, leading to heart and respiratory failure. A study on DMD mutant mice revealed that the mutation resulted in the upregulation of long noncoding RNAs (LncRNAs) and alterations in the metabolic pathway activity of endothelial cells (ECs). The findings provide new insights into the gene regulatory program in ECs in DMD and highlight the importance of further research in this area.
Review
Cardiac & Cardiovascular Systems
Satoshi Kawaguchi, Bruno Moukette, Taiki Hayasaka, Angela K. Haskell, Jessica Mah, Marisa N. Sepulveda, Yaoliang Tang, Il-man Kim
Summary: Noncoding RNAs are important in cardiac development and cardiovascular diseases. In this review, we discuss the critical roles of microRNAs, long noncoding RNAs, and circular RNAs in cardiac development and CVDs based on recent research articles. This review provides readers with a recent understanding of key ncRNAs and their mechanisms of action in cardiac development and CVDs.
JOURNAL OF CARDIOVASCULAR DEVELOPMENT AND DISEASE
(2023)
Article
Biochemistry & Molecular Biology
Yan Shen, Il-Man Kim, Yaoliang Tang
Summary: This study investigates the influence of dystrophin gene on the gene network of muscle stem cells. The mutation of dystrophin disrupts several long non-coding RNAs, affects cell adhesion, circulatory system process, and regulation of binding in muscle stem cells. Additionally, the metabolic pathway activity of muscle stem cells is altered.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Cell Biology
Varsha Tandra, Travis Anderson, Juan D. Ayala, Neal L. Weintraub, Nagendra Singh, Honglin Li, Jie Li
Summary: Protein modification by UFM1, called ufmylation, is important in regulating various physiological and pathological processes. UFBP1, the first identified ufmylation substrate, has been shown to play pivotal roles in development, hematopoiesis, intestinal homeostasis, chondrogenesis, and neuronal development. However, the significance of UFBP1 ufmylation in these processes is still unclear.
Article
Medicine, Research & Experimental
Tong Cheng, Zhusheng Chen, Yibin Qin, Xiang Zhu, Hongsheng Chen, Zhongling Xu, Xiaqing Ma
Summary: Morphine is commonly used and effective for pain relief, but its side effect of itching limits its clinical use. This paper discusses the potential of using esketamine to treat morphine-induced itching.
MEDICAL HYPOTHESES
(2024)
Article
Medicine, Research & Experimental
Sung Eun Lee, Eunjung Park, Ji-yun Kim, HyukHoon Kim
Summary: Hyperbaric oxygen therapy (HBOT) is a potential therapeutic modality that has been recognized for its favorable mechanisms in various diseases, including sepsis-associated encephalopathy (SAE). HBOT has neuroprotective effects through its anti-inflammatory and antiapoptotic effects as well as increased tissue oxygenation capacity. However, there are caveats and limitations in applying HBOT in sepsis.
MEDICAL HYPOTHESES
(2024)
