Article
Biotechnology & Applied Microbiology
Boram Son, Hyungro Yoon, Jina Ryu, Haein Lee, Jinmyoung Joo, Hee Ho Park, Tai Hyun Park
Summary: This study aimed to improve the efficiency of human iPSC generation by introducing the reprogramming factor Lin28. The researchers used a soluble Lin28-30Kc19 fusion protein to deliver Lin28 into the cells, resulting in an increased number and size of generated AP-positive hiPSC colonies. This recombinant protein could potentially be used as a valuable material for enhancing hiPSC generation efficiency and as an alternative to conventional methods.
FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY
(2022)
Review
Cell Biology
Heechang Moon, Bokwang Kim, Inbeom Kwon, Yohan Oh
Summary: Neurons derived from human pluripotent stem cells (hPSCs) are valuable for studying neural development and neurodegenerative diseases. Research on hPSC-based cell therapy for Parkinson's disease (PD) has shown promising results, but the lack of reliable lineage identification methods is a significant obstacle.
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY
(2023)
Article
Biochemistry & Molecular Biology
David A. Yefroyev, Sha Jin
Summary: Neurodegenerative diseases are severe pathologies that affect cognitive ability, motor control, and everyday function. Pluripotent stem cell technology offers hope in addressing this problem, with induced pluripotent stem cells providing an alternative and endless source without the ethical and practical concerns of embryonic stem cells. This review focuses on iPSC technology, the pathophysiology of Alzheimer's and Parkinson's diseases, and current state-of-the-art approaches using iPSCs for treatment.
Review
Cell & Tissue Engineering
Elena Coccia, Tim Ahfeldt
Summary: Research on Parkinson's disease has focused on the selective loss of dopaminergic neurons, with recent studies exploring both cell autonomous properties and non-cell autonomous contributions to the vulnerability of these neurons. These factors, including neuro-immune interactions, metabolic interactions, and damage to the vascular system, may collectively contribute to the specific vulnerability of these neurons and form a complex stressor-threshold model of neurodegeneration. Advances in modeling PD using human pluripotent stem cells, organoids, and CRISPR applications to edit PD genes aim to further understand the impact of genetic and environmental factors on the disease.
STEM CELL RESEARCH & THERAPY
(2021)
Review
Cell Biology
Xue Jiang, Yihuan Chen, Xiaofeng Liu, Lingqun Ye, Miao Yu, Zhenya Shen, Wei Lei, Shijun Hu
Summary: Researchers have discovered the contribution of genetic defects to the pathogenesis of primary cardiomyopathy and tried to explain the pathogenesis of these diseases by establishing various disease models. The advent of human induced pluripotent stem cells (hiPSCs) provides an unprecedented opportunity to further investigate the pathogenic mechanisms of inherited cardiomyopathies in vitro using patient-specific hiPSC-derived cardiomyocytes. The development of defects in genetically modified animal models differs notably from human diseases at the molecular level.
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY
(2021)
Article
Neurosciences
Ying Chen, Qing Yin, Xiao-Yu Cheng, Jin-Ru Zhang, Hong Jin, Kai Li, Cheng-Jie Mao, Fen Wang, Hong-Zhe Bei, Chun-Feng Liu
Summary: Induced pluripotent stem cells (iPSCs) can successfully mimic age-related diseases, such as Parkinson's disease (PD). In this study, we differentiated dopaminergic (DA) neurons from iPSCs of PD patients with the G2019S LRRK2 mutation and found morphological changes compared to control group. Furthermore, we observed that these mutated DA neurons were more vulnerable to damage, with higher rates of apoptosis and inflammatory response.
FRONTIERS IN NEUROSCIENCE
(2022)
Review
Immunology
Asuka Morizane
Summary: Parkinson's disease is the second most common neurodegenerative disease and a major target of cell therapies. Traditional cell therapies using aborted fetal tissue as donors face issues of donor material shortage, tissue quality instability, and ethical restrictions. However, advancements in stem cell technologies have allowed for the production of pluripotent stem cell-derived donor cells with unlimited scale, stable quality, and fewer ethical problems. Clinical trials of pluripotent stem cell-based therapies for Parkinson's disease have started based on positive results from non-clinical studies. This mini-review discusses practical considerations of stem cell-based therapies for Parkinson's disease, including different modes of transplantation and immune rejection.
INFLAMMATION AND REGENERATION
(2023)
Article
Multidisciplinary Sciences
Yu Jin Jang, Mijeong Kim, Bum-Kyu Lee, Jonghwan Kim
Summary: The authors developed a simple procedure to convert human primed pluripotent stem cells into trophoblast stem-like cells using bone morphogenetic protein 4, providing an important opportunity to study human placenta development and pathology.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
(2022)
Review
Biochemistry & Molecular Biology
Cameron L. McKnight, Yau Chung Low, David A. Elliott, David R. Thorburn, Ann E. Frazier
Summary: Mitochondrial diseases are a complex group of genetic disorders that disrupt cellular energy production, with no validated treatment options currently available. To study these diseases, many research groups have turned to using human pluripotent stem cells to model mitochondrial disease, allowing detailed investigation of cellular pathomechanisms and validation of potential treatment options.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Cell Biology
Glyn Nigel Stacey, Jie Hao
Summary: In recent years, significant progress has been made in China in establishing hPSCs for the manufacture of cell therapies. The National Stem Cell Resource Center and its partner organizations have over 600 hPSC lines formally recorded, with 47 of them also registered for European Commission funded research projects. This national and international coordination aims to promote the translation of Chinese hPSC-based products into clinical use according to established standards.
CELL PROLIFERATION
(2022)
Article
Cell Biology
Alexandra Neaverson, Malin H. L. Andersson, Osama A. Arshad, Luke Foulser, Mary Goodwin-Trotman, Adam Hunter, Ben Newman, Minal Patel, Charlotte Roth, Tristan Thwaites, Helena Kilpinen, Matthew E. Hurles, Andrew Day, Sebastian S. Gerety
Summary: Efficient and effective methods for converting human induced pluripotent stem cells into differentiated derivatives are critical for performing robust, large-scale studies of development and disease modelling, and for providing a source of cells for regenerative medicine. Here, we describe a 14-day neural differentiation protocol which allows for the scalable, simultaneous differentiation of multiple iPSC lines into cortical neural stem cells.
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY
(2023)
Article
Cell & Tissue Engineering
Nayeon Lee, Hye-Ji Moon, So-Hyun Park, Ji-Young Moon, Ka-Kyung Park, Jae-Ho Kim, Jae-Hyeok Lee
Summary: The hiPSC line was generated from a male patient with young onset Parkinson's disease, carrying a heterozygous c.680 A > G (N227S) mutation in the GBA gene. The reprogrammed hiPSC line displayed a normal karyotype and expression of pluripotency markers, capable of producing derivatives of three germ layers.
STEM CELL RESEARCH
(2022)
Article
Cell Biology
Warunya Chakritbudsabong, Somjit Chaiwattanarungruengpaisan, Ladawan Sariya, Sirikron Pamonsupornvichit, Joao N. Ferreira, Panithi Sukho, Dulyatad Gronsang, Theerawat Tharasanit, Andras Dinnyes, Sasitorn Rungarunlert
Summary: In addition to the four transcription factors, LIN28 is required for the effective induction of piPSCs and the maintenance of their long-term self-renewal and pluripotency towards the development of all germ layers.
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY
(2021)
Article
Cell Biology
Ying Zhang, Jun Wei, Jiani Cao, Kehua Zhang, Yaojin Peng, Hongkui Deng, Jiuhong Kang, Guangjin Pan, Yong Zhang, Boqiang Fu, Shijun Hu, Jie Na, Yan Liu, Lei Wang, Lingmin Liang, Huanxin Zhu, Yu Zhang, Zi-Bing Jin, Jie Hao, Aijin Ma, Tongbiao Zhao, Junying Yu
Summary: "Requirements for Human-Induced Pluripotent Stem Cells" is the first set of guidelines in China on human-induced pluripotent stem cells. It provides detailed technical requirements and aims to promote international standardization.
CELL PROLIFERATION
(2022)
Article
Developmental Biology
K. Lundin, K. Sepponen, P. Vayrynen, X. Liu, D. A. Yohannes, M. Survila, B. Ghimire, J. Kansakoski, S. Katayama, J. Partanen, S. Vuoristo, P. Paloviita, N. Rahman, T. Raivio, K. Luiro, I Huhtaniemi, M. Varjosalo, T. Tuuri, J. S. Tapanainen
Summary: This study investigates the function of follicle-stimulating hormone (FSH) and its receptor (FSHR) using in vitro models. By differentiating FSHR-expressing cells from human pluripotent stem cells, the researchers were able to study endogenous FSHR function. The results show that FSH stimulation activates the cAMP signaling pathway and downstream mediators, and the analysis of protein-protein interactions revealed key proteins associated with infertility.
MOLECULAR HUMAN REPRODUCTION
(2022)
Review
Clinical Neurology
Se Jin Cho, Byung Se Choi, Yun Jung Bae, Jae-Jin Song, Ja-Won Koo, Ji-Soo Kim, Sung Hyun Baik, Leonard Sunwoo, Jae Hyoung Kim
Summary: This systematic review evaluated the diagnostic performance of MRI for intralabyrinthine schwannoma (ILS) patients. The combination of T2WI and CE-T1WI showed high sensitivity and specificity for diagnosing ILS. However, further research is needed to reduce heterogeneity among studies.
JOURNAL OF NEURORADIOLOGY
(2022)
Article
Medicine, General & Internal
Liping Lan, Yin Liu, Yuanqing Wu, Zhen-Gui Xu, Jin-Jing Xu, Jae-Jin Song, Richard Salvi, Xindao Yin, Yu-Chen Chen, Yuexin Cai
Summary: This study examines the role of aberrant brain networks in tinnitus treatment. The findings suggest that the effectiveness of neuromodulation with repetitive transcranial magnetic stimulation (rTMS) and sound therapy utilizing tailor-made notch music training (TMNMT) varies depending on the functional brain network of the patients.
Article
Biochemistry & Molecular Biology
Hyeji Park, Tae-In Kam, Hanjing Peng, Shih-Ching Chou, Amir A. Mehrabani-Tabari, Jae-Jin Song, Xiling Yin, Senthilkumar S. Karuppagounder, George K. Umanah, A. V. Subba Rao, YuRee Choi, Akanksha Aggarwal, Sohyun Chang, Hyunhee Kim, Jiyoung Byun, Jun O. Liu, Ted M. Dawson, Valina L. Dawson
Summary: The study reveals that Parthanatos-associated apoptosis-inducing factor can induce neurodegeneration through nuclease activity, and an inhibitor called PAANIB-1 can prevent this degeneration, which is of broad importance in developing cell death inhibitors targeting the druggable PAAN/MIF nuclease in human pathologies.
Article
Gastroenterology & Hepatology
Ju Hee Oh, Dae Won Jun, Hye Young Kim, Seung Min Lee, Eileen L. Yoon, Jungwook Hwang, Jung Hwan Park, Hanbi Lee, Wankyu Kim, Hyunsung Kim
Summary: This study aimed to identify an optimal target population and drug-specific biomarkers that can predict dipeptidyl peptidase (DPP)-4 inhibitor responses in non-alcoholic fatty liver disease (NAFLD). The study found that DPP-4 inhibitors attenuated NAFLD activity score and fibrosis stage in the high-fat diet (HFD) model. Additionally, hepatic or serum levels of Igfbp-1 were associated with DPP-4 inhibitor response.
CLINICAL AND MOLECULAR HEPATOLOGY
(2022)
Review
Ophthalmology
Dong Hyun Jo, Sangsu Bae, Hyongbum Henry Kim, Jin-Soo Kim, Jeong Hun Kim
Summary: Inherited retinal diseases (IRDs) are vision-threatening retinal disorders caused by gene variants related to vision. Current treatment options are limited, but genome editing using CRISPR-Cas9 technologies shows promise for correcting pathogenic variants and providing new treatment opportunities.
PROGRESS IN RETINAL AND EYE RESEARCH
(2023)
Article
Biochemistry & Molecular Biology
Goosang Yu, Hui Kwon Kim, Jinman Park, Hyunjong Kwak, Yumin Cheong, Dongyoung Kim, Jiyun Kim, Jisung Kim, Hyongbum Henry Kim
Summary: In this study, the efficiency of prime editing was evaluated for a large number of pegRNAs. Computational models, DeepPrime and DeepPrime-FT, were developed to predict editing efficiencies for different prime editing systems and cell types. The study also included profiling of editing efficiencies at mismatched targets and the development of a computational model for predicting editing efficiencies at such targets. These findings and models will greatly enhance the applications of prime editing.
Correction
Biochemistry & Molecular Biology
Yunseon Yang, Min-Jong Seok, Ye Eun Kim, Yunjung Choi, Jae-Jin Song, Yanuar Alan Sulistio, Seong-hoon Kim, Mi-Yoon Chang, Soo-Jin Oh, Min-Ho Nam, Yun Kyung Kim, Tae-Gyun Kim, Heh-In Im, Seong-Ho Koh, Sang-Hun Lee
MOLECULAR PSYCHIATRY
(2023)
Article
Biotechnology & Applied Microbiology
Nahye Kim, Sungchul Choi, Sungjae Kim, Myungjae Song, Jung Hwa Seo, Seonwoo Min, Jinman Park, Sung-Rae Cho, Hyongbum Henry Kim
Summary: A deep learning model is used to predict the best base editor for specific applications. Seven base editors and nine Cas9 variants were systematically compared to determine their editing windows, outcomes, and preferred motifs. Two computational models, DeepCas9variants and DeepBE, were developed to predict the efficiency and outcomes of base editors, resulting in significantly higher efficiency for DeepBE-designed editors compared to rational design.
NATURE BIOTECHNOLOGY
(2023)
Article
Biochemistry & Molecular Biology
Young-hoon Kim, Nahye Kim, Ikenna Okafor, Sungchul Choi, Seonwoo Min, Joonsun Lee, Seung-Min Bae, Keunwoo Choi, Janice Choi, Vinayak Harihar, Youngho Kim, Jin-Soo Kim, Benjamin P. Kleinstiver, Jungjoon K. Lee, Taekjip Ha, Hyongbum Henry Kim
Summary: Researchers have developed an improved version of Sniper-Cas9, called Sniper2L, which combines high specificity with retained high activity for efficient and specific genome editing at a large number of target sequences.
NATURE CHEMICAL BIOLOGY
(2023)
Article
Biochemical Research Methods
Sang-Yeon Seo, Seonwoo Min, Sungtae Lee, Jung Hwa Seo, Jinman Park, Hui Kwon Kim, Myungjae Song, Dawoon Baek, Sung-Rae Cho, Hyongbum Henry Kim
Summary: This study compares the effectiveness of 17 small Cas9s and provides guidance for researchers to select the most suitable small Cas9 for specific target sequences.
Article
Chemistry, Medicinal
Gi-Ae Kim, Hyun Chin Cho, Soung Won Jeong, Bo-Kyeong Kang, Mimi Kim, Seungwon Jung, Jungwook Hwang, Eileen L. Yoon, Dae Won Jun
Summary: Preclinical data have shown that ALS-L1023, an herbal extract from Melissa officinalis, can reduce visceral fat and hepatic steatosis. In this study, we evaluated the safety and efficacy of ALS-L1023 as a treatment for non-alcoholic fatty liver disease (NAFLD). The results showed that ALS-L1023 could significantly reduce hepatic fat content and improve liver stiffness in patients with NAFLD, with no significant differences in adverse events among the study groups.
Article
Biology
Jung Hwa Seo, Jeong Hong Shin, Junwon Lee, Daesik Kim, Hye-Yeon Hwang, Bae-Geun Nam, Jinu Lee, Hyongbum Henry Kim, Sung-Rae Cho
Summary: The use of CRISPR interference (CRISPRi) can repress the expression of mutant huntingtin while preserving wild-type huntingtin, potentially delaying disease progression in Huntington's disease, which is caused by a CAG repeat expansion in the huntingtin gene.
COMMUNICATIONS BIOLOGY
(2023)
Correction
Biochemistry & Molecular Biology
Yunseon Yang, Min-Jong Seok, Ye Eun Kim, Yunjung Choi, Jae-Jin Song, Yanuar Alan Sulistio, Seong-hoon Kim, Mi-Yoon Chang, Soo-Jin Oh, Min-Ho Nam, Yun Kyung Kim, Tae-Gyun Kim, Heh-In Im, Seong-Ho Koh, Sang-Hun Lee
MOLECULAR PSYCHIATRY
(2023)
Article
Cell & Tissue Engineering
Jin Saem Lee, Jeoung Eun Lee, Shin-Hye Yu, Taehoon Chun, Mi-Yoon Chang, Dong Ryul Lee, Chang-Hwan Park
Summary: Human pluripotent stem cells have potential in cell therapy, but face challenges in clinical trials due to immune rejection. Homozygous SCNT-hESCs simplify clinical application and reduce HLA mismatch. This study shows no differences in differentiation capacity and HLA expression among hPSCs, suggesting wider applications for homo-SCNT-hESCs in transplants with neural precursor cells.
INTERNATIONAL JOURNAL OF STEM CELLS
(2023)