Advanced In Vitro Models for Preclinical Drug Safety: Recent Progress and Prospects by Dileep G. Nair, Ralf Weiskirchen

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Integrating pharmacogenetics, multi‐omics and machine learning in the novel therapeutic view of pulmonary hypertension by Chenxi Li, Jipeng Xue, Dongchen Zhou, Xiaomeng Qi, Ting Chen, Zhoubin Li

“…In addition to the commonly used drugs for treating PH, pharmacogenomic therapies are facilitating the innovation of personalized medicine to treat PH. Meanwhile, gene therapy has been extensively studied but is a quite challenging task. …”
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Induced Pluripotent Stem Cells and Outer Retinal Disease by Jin Yang, Bingcui Cai, Patrick Glencer, Zhiqing Li, Xiaomin Zhang, Xiaorong Li

“…The retina, which is composed of multiple layers of differing cell types, has been considered the first choice for gene therapy, disease modeling, and stem cell-derived retinal cell transplant therapy. …”
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Three-dimensional linkage analysis with digital PCR for genome integrity and identity of recombinant adeno-associated virus by Tam Duong, Michele Firmo, Chien-Ting Li, Bingnan Gu, Peng Wang

“…Utilizing rAAV in gene therapy requires a highly precise quantification method to determine the viral genome titer and further establish the optimal therapeutic dosage for a rAAV product. …”
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Study on Apoptosis of Prostate Cancer Cells Induced by IκBα Overexpression Synergistic with Poly (Lactic-Co-Glycolic Acid)-Curcumin Nanoparticles by Hui Guo, Jianwen Huang, Hongbin Li, Ranxing Yang, Rong Chen

“…These findings may provide an experimental basis to establish the tumor gene therapy combined with traditional Chinese medicine treatment, thus promoting the clinical application of both tumor gene therapy and anti-tumor Chinese medicine.…”
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Topic “Signaling Pathways in Liver Disease” by Ralf Weiskirchen

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Pancreatic organogenesis mapped through space and time by Marissa A. Scavuzzo, Wojciech J. Szlachcic, Matthew C. Hill, Natalia M. Ziojla, Jessica Teaw, Jeffrey C. Carlson, Jonathan Tiessen, Jolanta Chmielowiec, James F. Martin, Malgorzata Borowiak

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A Full GMP Process to Select and Amplify Epitope-Specific T Lymphocytes for Adoptive Immunotherapy of Metastatic Melanoma by N. Labarriere, A. Fortun, A. Bellec, A. Khammari, B. Dreno, S. Saïagh, F. Lang

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Engineering Cell Systems by Tiago G. Fernandes, Ricardo P. Baptista, Howard Kim

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Survival, Incidence, and Mortality Trends in Female Cancers in the Nordic Countries by Filip Tichanek, Asta Försti, Otto Hemminki, Akseli Hemminki, Kari Hemminki

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Parental perception of treatment options for mucopolysaccharidosis: a survey to bridge the gap for personalized medicine by Anna-Maria Wiesinger, Florian B. Lagler

“…While over 80% of respondents who held a positive attitude toward ERT, HSCT, and ITTs indicated they would opt for these therapies in both neuronopathic and non-neuronopathic cases, only about half of the parents with a favorable general view of gene therapy (GT) expressed a positive perception of its likelihood as a treatment option. …”
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Development of cell lines with increased susceptibility to diverse adeno-associated viral vectors to enable in vitro potency assays by James Zengel, Emma S. Esterman, Anitha Ponnuswami, Nicholas R. Wall, Jan E. Carette

“…Vectors based on adeno-associated viruses (AAVs) are promising therapeutic modalities used in gene therapy. Robust cell-based assays that demonstrate and quantify the potency of AAV vectors in expressing their transgene are needed for clinical development. …”
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Novel banana lectin CAR-T cells to target pancreatic tumors and tumor-associated stroma by Malcolm K Brenner, Challice Bonifant, Norihiro Watanabe, Mary K McKenna, Daniel Brenner, Ada Ozcan, Maureen Legendre, Dafydd G Thomas, Christopher Ashwood, Richard D Cummings, David M Markovitz

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The Immunomodulatory Properties of Mesenchymal Stem Cells by Junji Xu, Dunfang Zhang, Peter Zanvit, Dandan Wang

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iTRAQ-Based Proteomic Analysis of Visual Cycle-Associated Proteins in RPE of rd12 Mice before and after RPE65 Gene Delivery by Qinxiang Zheng, Yueping Ren, Radouil Tzekov, Shanshan Hua, Minghan Li, Jijing Pang, Jia Qu, Wensheng Li

“…RALBP-1, RBP-1, and IRBP were reduced in the untreated rd12 eyes and the former two were improved after gene therapy, confirmed by Western blot analysis. Conclusions. …”
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DNA-mediated self-assembly oxidative damage amplifier combined with copper and MTH1 inhibitor for cancer therapy by Cui Ren, Zhiyong Shi, Xiaowen Zhang, Xueer Yu, Yang Gao, Zhi Qi, Yu Chen, Yong Wang

“…In this research, DNA-mediated self-assembly nanoparticles (HTCG@TA NPs) were engineered with high-performance amplified oxidative damage and gene therapy effect for synergistic anti-tumor treatment. …”
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The Degeneration and Apoptosis Patterns of Cone Photoreceptors in rd11 Mice by Hua Zhang, Xia Li, Xufeng Dai, Juanjuan Han, Yangyang Zhang, Yan Qi, Ying He, Yan Liu, Bo Chang, Ji-jing Pang

“…These indicated that the degeneration and apoptosis of cone photoreceptors began at P14 of rd11 mice, which was a key point for gene therapy.…”
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Robust genome editing activity and the applications of enhanced miniature CRISPR-Cas12f1 by Soo-Ji Park, Sungjin Ju, Won Jun Jung, Tae Yeong Jeong, Da Eun Yoon, Jang Hyeon Lee, Jiyun Yang, Hojin Lee, Jungmin Choi, Hyeon Soo Kim, Kyoungmi Kim

“…Furthermore, we demonstrate that eCas12f1 efficiently induces apoptosis in cancer cells and is compatible with base editing and regulation of gene expression, verifying its high utility and applicability in gene therapy research.…”
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Role of cardiolipin in skeletal muscle function and its therapeutic implications by Youngbum Yoo, MyeongHoon Yeon, Mee-Sup Yoon, Young-Kyo Seo

“…Therapeutic strategies, including CL-targeting compounds like elamipretide and gene therapy, show promise for mitigating sarcopenia by improving mitochondrial bioenergetics and muscle regeneration.…”
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