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  1. No Access

    Article

    Stable inhibition of choroidal neovascularization by adeno-associated virus 2/8-vectored bispecific molecules

    Neovascular age-related macular degeneration (nAMD) causes severe visual impairment. Pigment epithelium-derived factor (PEDF), soluble CD59 (sCD59), and soluble fms-like tyrosine kinase-1 (sFLT-1) are potentia...

    Tinghui Bai, Bohao Cui, Man **ng, Siyue Chen, Yanfang Zhu, Dongxue Lin in Gene Therapy (2024)

  2. No Access

    Article

    PARP inhibitor and immune checkpoint inhibitor have synergism efficacy in gallbladder cancer

    Gallbladder cancer (GBC) is an aggressive cancer with poor prognosis. PARP inhibitors (PARPi) target PARP enzymes and have shown efficacy in patients with breast cancer gene (BRCA) mutations. Immunotherapy, es...

    Yu Chen, Xudong Fan, Ruohuang Lu, Shan Zeng, **** Gan in Genes & Immunity (2024)

  3. Article

    Open Access

    Retinoic acid related orphan receptor α is a genetic modifier that rescues retinal degeneration in a mouse model of Stargardt disease and Dry AMD

    Degeneration of the macula is associated with several overlap** diseases including age-related macular degeneration (AMD) and Stargardt Disease (STGD). Mutations in ATP Binding Cassette Subfamily A Member 4 (AB...

    M. Akula, S. M. McNamee, Z. Love, N. Nasraty, N. P. M. Chan, M. Whalen in Gene Therapy (2024)

  4. Article

    Open Access

    Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates

    In this study, we demonstrate the safety and utility of CRISPR-Cas9 gene editing technology for in vivo editing of proviral DNA in ART-treated, virally controlled simian immunodeficiency virus (SIV) infected r...

    Tricia H. Burdo, Chen Chen, Rafal Kaminski, Ilker K. Sariyer in Gene Therapy (2024)

  5. No Access

    Article

    CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy

    EPM1 is the most common form of Progressive Myoclonus Epilepsy characterized by late-childhood onset, ever-worsening and disabling myoclonus, seizures, ataxia, psychiatric disease, and shortened lifespan. EPM1...

    Emrah Gumusgoz, Sahba Kasiri, Mayank Verma, Jun Wu, Daniel Villarreal Acha in Gene Therapy (2024)

  6. Article

    Open Access

    A multinational survey of potential participant perspectives on ocular gene therapy

    Amidst rapid advancements in ocular gene therapy, understanding patient perspectives is crucial for sha** future treatment choices and research directions. This international cross-sectional survey evaluated...

    Alexis Ceecee Britten-Jones, Myra B. McGuinness, Fred K. Chen in Gene Therapy (2024)

  7. No Access

    Article

    Pharmacogenomic study—A pilot study of the effect of pharmacogenomic phenotypes on the adequate dosing of verapamil for migraine prevention

    To investigate factors affecting the efficacy and tolerability of verapamil for migraine prevention using individual pharmacogenomic phenotypes.

    Yi-Chieh Chen, Han Wang, Jayawant N. Mandrekar in The Pharmacogenomics Journal (2024)

  8. Article

    Open Access

    Pharmacogenetic and clinical risk factors for bevacizumab-related gastrointestinal hemorrhage in prostate cancer patients treated on CALGB 90401 (Alliance)

    The objective of this study was to discover clinical and pharmacogenetic factors associated with bevacizumab-related gastrointestinal hemorrhage in Cancer and Leukemia Group B (Alliance) 90401. Patients with m...

    Jai N. Patel, Chen Jiang, Kouros Owzar, Daniel L. Hertz in The Pharmacogenomics Journal (2024)

  9. Article

    Retraction Note: Long noncoding RNA RBMS3-AS3 acts as a microRNA-4534 sponge to inhibit the progression of prostate cancer by upregulating VASH1

    Zhenming Jiang, Yuxi Zhang, ** Chen, **eng Wu, Dong Chen in Gene Therapy (2024)

  10. Article

    Open Access

    Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates

    Tricia H. Burdo, Chen Chen, Rafal Kaminski, Ilker K. Sariyer in Gene Therapy (2024)

  11. Article

    Open Access

    Successes and challenges in clinical gene therapy

    Despite the ups and downs in the field over three decades, the science of gene therapy has continued to advance and provide enduring treatments for increasing number of diseases. There are active clinical tria...

    Donald B. Kohn, Yvonne Y. Chen, Melissa J. Spencer in Gene Therapy (2023)

  12. No Access

    Article

    Knowledge and attitudes of medical and pharmacy students about pharmacogenomics: a systematic review and meta-analysis

    Pharmacogenomics (PGx) is rapidly growing branch of molecular genetics with high potentials to influence therapeutics. This review evaluates knowledge and attitudes of medical and pharmacy students about PGx. ...

    Chen Li, **aona Su, Qidi Sun, Yi Huang in The Pharmacogenomics Journal (2023)

  13. Article

    Retraction Note: Inhibition of RM-1 prostate carcinoma and eliciting robust immune responses in the mouse model by using VEGF-M2-GnRH3-hinge-MVP vaccine

    Yiqin Wang, Murad Alahdal, Jia Ye, Liangliang **g, **aoxin Liu in Genes & Immunity (2023)

  14. No Access

    Article

    Integrated analyses reveal the prognostic, immunological features and mechanisms of cuproptosis critical mediator gene FDX1 in KIRC

    The ferredoxin 1 (FDX1) gene had been recently reported as a critical mediator of cuproptosis, and without doubt, its roles in KIRC would be of importance. Hence, this paper was to explore the roles of FDX1 in...

    Yi Wang, **nyu Zhang, Guihua Chen, Qianwei **ng, Bingye Zhu, **ang Wang in Genes & Immunity (2023)

  15. Article

    Open Access

    Potential diagnostic biomarkers: 6 cuproptosis- and ferroptosis-related genes linking immune infiltration in acute myocardial infarction

    The current diagnostic biomarkers of acute myocardial infarction (AMI), troponins, lack specificity and exist as false positives in other non-cardiac diseases. Previous studies revealed that cuproptosis, ferro...

    Mengdan Miao, Shanhu Cao, Yifei Tian, Da Liu, Lixia Chen, Qiaoying Chai in Genes & Immunity (2023)

  16. No Access

    Article

    Suppression of bone metastatic castration-resistant prostate cancer cell growth by a suicide gene delivered by JC polyomavirus-like particles

    Prostate cancer is one of the most common cancers in men. The heterogeneity and mutations exhibited by prostate cancer cells often results in the progression to incurable metastatic castration-resistant prosta...

    Cheng-Huang Shen, Mien-Chun Lin, Chiung-Yao Fang, Pei-Lain Chen in Gene Therapy (2023)

  17. Article

    Open Access

    Identification of heat shock protein family A member 5 (HSPA5) targets involved in nonalcoholic fatty liver disease

    Heat shock protein family A (Hsp70) member 5 (HSPA5) is an endoplasmic reticulum chaperone, which regulates cell metabolism, particularly lipid metabolism. While HSPA5’s role in regulating cell function is wel...

    Aliya Rehati, Buzukela Abuduaini, Zhao Liang, Dong Chen, Fang** He in Genes & Immunity (2023)

  18. Article

    Open Access

    αCGRP deficiency aggravates pulmonary fibrosis by activating the PPARγ signaling pathway

    In order to explore whether αCGRP (Calca) deficiency aggravates pulmonary fibrosis (PF). Clinical data from patients with PF (n = 52) were retrospectively analyzed. Lung tissue from a bleomycin (BLM)-induced rat ...

    **aoting Lv, Qingquan Chen, Zewei Zhang, Kaili Du, Ya** Huang in Genes & Immunity (2023)

  19. No Access

    Article

    Investigating genetic variants for treatment response to selective serotonin reuptake inhibitors in syndromal factors and side effects among patients with depression in Taiwanese Han population

    Major depressive disorder (MDD) is associated with high heterogeneity in clinical presentation. In addition, response to treatment with selective serotonin reuptake inhibitors (SSRIs) varies considerably among...

    Shiau-Shian Huang, Yi-Ting Chen, Mei-Hsin Su, Shih-Jen Tsai in The Pharmacogenomics Journal (2023)

  20. No Access

    Article

    Distinct functions of CAR-T cells possessing a dectin-1 intracellular signaling domain

    Chimeric antigen receptor T (CAR-T) cell therapy has demonstrated remarkable efficacies in treating hematopoietic malignancies, but not in the solid tumors. Incorporating costimulatory signaling domains, such ...

    **ao Liang, Yong Huang, Dan Li, **ao Yang, Lin Jiang, Weilin Zhou in Gene Therapy (2023)

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