Gene therapy and its applications by Alka Bansal, Ravi Prakash, Swati Agarwal, Uma Advani

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Gene Therapy for Primary Immunodeficiency by Benjamin C. Houghton, Claire Booth

“…Here, we review the key developments in the gene therapy landscape for primary immune deficiency, from an experimental therapy where clinical efficacy was marred by adverse events, to a commercialized product with enhanced safety and efficacy. …”
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Gene therapy in neuromuscular disorders by Rodrigo Holanda Mendonça, Edmar Zanoteli

“…Abstract Monogenic neuromuscular disorders are potentially treatable through gene therapy. Using viral vectors, a therapeutic transgene aims to restore normal levels of a protein not produced by the defective gene, or to silence a gene whose expression leads to toxic effects. …”
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Gene Therapy in Pediatric Orthopedics by Emmanuel Olaonipekun, Anthony Lisyansky, Robin Olaonipekun, Bouchra Ghania Merabia, Karim Gaber, Waleed Kishta

Subjects: “…gene therapy…”
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Gene Therapy Today and Tomorrow by Kenneth Lundstrom

“…In the wake of a breakthrough in biotechnology providing realistic application of recombinant expressed proteins as drugs in the 1990s, gene therapy emerged as the potential approach for providing medicines of the future [...]…”
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Gene Therapy to the Retina and the Cochlea by Ryan Crane, Shannon M. Conley, Shannon M. Conley, Muayyad R. Al-Ubaidi, Muayyad R. Al-Ubaidi, Muayyad R. Al-Ubaidi, Muna I. Naash, Muna I. Naash, Muna I. Naash

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Gene Therapy for Glioblastoma Multiforme by Smit Shah, Joshua Green, Shantelle A. Graff, Qi Li, John D. Heiss

“…The possible benefits of combining gene therapy with standard GBM treatments will also be covered.…”
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Biomaterial‐based gene therapy by Yi Yu, Yijun Gao, Liming He, Bairong Fang, Wenhui Ge, Pu Yang, Yikun Ju, Xiaoyan Xie, Lanjie Lei

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Viral Vectors in Gene Therapy by Kenneth Lundstrom

“…Applications of viral vectors have found an encouraging new beginning in gene therapy in recent years. Significant improvements in vector engineering, delivery, and safety have placed viral vector-based therapy at the forefront of modern medicine. …”
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Switching on the lights for gene therapy. by Alexandra Winkeler, Miguel Sena-Esteves, Leonie E M Paulis, Hongfeng Li, Yannic Waerzeggers, Benedikt Rückriem, Uwe Himmelreich, Markus Klein, Parisa Monfared, Maria A Rueger, Michael Heneka, Stefan Vollmar, Mathias Hoehn, Cornel Fraefel, Rudolf Graf, Klaus Wienhard, Wolf D Heiss, Andreas H Jacobs

“…Non-invasive assessment of the dynamics of gene regulation is of interest for the detection of endogenous disease-specific biological alterations (e.g., signal transduction) and for monitoring the induction and regulation of therapeutic genes (e.g., gene therapy). To demonstrate that non-invasive imaging of regulated expression of any type of gene after in vivo transduction by versatile vectors is feasible, we generated regulatable herpes simplex virus type 1 (HSV-1) amplicon vectors carrying hormone (mifepristone) or antibiotic (tetracycline) regulated promoters driving the proportional co-expression of two marker genes. …”
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Advances in Cancer Gene Therapy by Ming Yu

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Gene therapy for retinitis pigmentosa by Niloofar Piri, Jacob D Grodsky, Henry J Kaplan

Subjects: “…gene therapy…”
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Gene Therapy for Bone Engineering by Elizabeth eRosado Balmayor, Elizabeth eRosado Balmayor, Martijn eVan Griensven

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Gene therapy for retinal diseases by Nasrollah Samiy

“…Gene therapy has tremendous potential for different ocular diseases. …”
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Gene Therapy for Regenerative Medicine by Hossein Hosseinkhani, Abraham J. Domb, Ghorbanali Sharifzadeh, Victoria Nahum

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Safety issues of gene therapy by S. E. Ipatov, S. A. Roumiantsev

Subjects: “…gene therapy…”
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The once and future gene therapy by Karen Bulaklak, Charles A. Gersbach

“…Gene therapy is at an inflection point. Recent successes in genetic medicine have paved the path for a broader second wave of therapies and laid the foundation for next-generation technologies. …”
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GENE THERAPY IN THALASSEMIA AND HEMOGLOBINOPATHIES by Laura Breda, Roberto Gambari, Stefano Rivella

“…This review discusses gene therapy strategies for the hemoglobinopathies, including the use of lentiviral vectors, generation of induced pluripotent stem cells (iPS) cells, gene targeting, splice-switching and stop codon readthrough.…”
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Gene therapy for colorectal cancer. by Kerr, D, Seymour, L, Maruta, F

“…Although the preclinical results of gene therapy for colorectal cancer have shown promise, gene therapy is still at an early stage of clinical development and has not yet shown a significant therapeutic benefit for patients. …”

Gene therapy matures in the clinic. by Seymour, L, Thrasher, A