Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy by Craig M. McDonald, Jacob S. Elkins, Sai Dharmarajan, Katherine Gooch, Teofil Ciobanu, Claire J. Lansdall, Alexander P. Murphy, Fiona McDougall, Eugenio M. Mercuri, Ivana Audhya, the EMBARK Study Group

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A Korean Patient With Leber Congenital Amaurosis and a Homozygous RPE65 Variant Originating From a Paternal Uniparental Isodisomy by Hane Lee, Dongseok Moon, Rin Khang, Go Hun Seo, Chang Ki Yoon, Un Chul Park, Kyu Hyung Park, Eun Kyoung Lee

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CRISPR screen reveals modifiers of rAAV production including known rAAV infection genes playing an unexpected role in vector production by Emily E. O’Driscoll, Sakshi Arora, Jonathan F. Lang, Beverly L. Davidson, Ophir Shalem

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Development of the Dutch translational knowledge agenda for inherited metabolic diseases by I. J. Hieltjes, J. H. van derLee, M. C. Groenendijk, G. vanHaaften, P. M. vanHasselt, R. J. Lunsing, G. J. J. vanProoijen, E. M. deRuiter, F. J. vanSpronsen, N. M. Verhoeven‐Duif, A. deVreugd, M. Wagenmakers, H. Zweers, H. Dekker, H. R. Waterham, C. D. vanKarnebeek, R. J. A. Wanders, R. A. Wevers

Subjects: “…gene therapy…”
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Gene Therapy with HSV1-sr39TK/GCV Exhibits a Stronger Therapeutic Efficacy Than HSV1-TK/GCV in Rat C6 Glioma Cells by Lei-qing Li, Fang Shen, Xiao-yan Xu, Hong Zhang, Xiao-feng Yang, Wei-guo Liu

“…Finally, a subcutaneously C6 xenograft tumor model was established in the nude mice to test the in vitro efficacy of TK/GCV gene therapy. Our results showed that, as compared with wildtype TK, HSV1-sr39TK/GCV demonstrated a stronger therapeutic efficacy against C6 glioma both in vitro and in vivo, which, by reducing the required GCV dose, might warrant its future use in the treatment of glioma under clinical setting.…”
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mTOR downregulation promotes anti-inflammatory responses via the CCL3-CCR5 axis in hypoxic retinopathy by Tae Kwon Moon, Im Kyeung Kang, Kyoung Jin Lee, Ji Hyun Kim, Hee Jong Kim, A. Reum Han, Ha-Na Woo, Joo Yong Lee, Jun-Sub Choi, Keerang Park, Heuiran Lee

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Emerging Strategies in Cartilage Repair and Joint Preservation by Mircea Adrian Focsa, Sorin Florescu, Armand Gogulescu

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Neuromuscular diseases: genomics-driven advances by Anna Cho

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Progress in photoreceptor replacement therapy for retinal degenerative diseases by Yuxin Du, Yin Shen

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Efficient Dual Cas9 Nickase Correction of a Prevalent Pathogenic LAMB3 Variant for Junctional Epidermolysis Bullosa by Alex du Rand, John Hunt, Daniel Verdon, Ben Buttle, P. Rod Dunbar, Diana Purvis, Vaughan Feisst, Hilary Sheppard

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Glutamine missense suppressor transfer RNAs inhibit polyglutamine aggregation by Rasangi Tennakoon, Teija M.I. Bily, Farah Hasan, Sunidhi Syal, Aaron Voigt, Tugce B. Balci, Kyle S. Hoffman, Patrick O’Donoghue

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Clinically meaningful improvements after gene therapy for aromatic L-amino acid decarboxylase deficiency (AADCd) in the Peabody Developmental Motor Scale, Second Edition (PDMS-2) and correlation with Bayley-III scores and motor milestones by Wuh-Liang Hwu, Hui-Min Lee, John Devin Peipert, Rongrong Zhang, Christian Werner, J. Rafael Sierra, Thomas O’Connell, Jonathan J. Woolley, Marjorie Crowell, Antonia Wang, Ioannis Tomazos

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The Genetic Basis of Congenital Cataracts: Advances in Diagnostics and Therapeutics by Agnieszka Starzyk, Piotr Charzewski

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Silencing hepatic PCSK9 via novel chimeric AAV8 mitigates the progression of atherosclerosis by inhibiting inflammation in ApoE−/− mice by Xiaocui Chen, Minghui Sun, Xiang Ma, Yitong Ma, Bangdang Chen

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Therapeutic landscape for inherited ocular diseases: current and emerging therapies by Hwei Wuen Chan, Jaslyn Oh, Bart Leroy

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Advancing CNS Therapeutics: Enhancing Neurological Disorders with Nanoparticle-Based Gene and Enzyme Replacement Therapies by Liu S, Li H, Xi S, Zhang Y, Sun T

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NPRL2 gene therapy induces effective antitumor immunity in KRAS/STK11 mutant anti-PD1 resistant metastatic non-small cell lung cancer (NSCLC) in a humanized mouse model by Ismail M Meraz, Mourad Majidi, Renduo Song, Feng Meng, Lihui Gao, Qi Wang, Jing Wang, Elizabeth J Shpall, Jack A Roth

Subjects: “…nprl2 gene therapy…”
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Harnessing the AI/ML in Drug and Biological Products Discovery and Development: The Regulatory Perspective by Fahimeh Mirakhori, Sarfaraz K. Niazi

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Vector‐Free Deep Tissue Targeting of DNA/RNA Therapeutics via Single Capacitive Discharge Conductivity‐Clamped Gene Electrotransfer by Jeremy L. Pinyon, Georg vonJonquieres, Stephen L. Mow, Amr Al Abed, Keng‐Yin Lai, Mathumathi Manoharan, Edward N. Crawford, Stanley H. Xue, Sarah Smith‐Moore, Lisa J. Caproni, Sarah Milsom, Matthias Klugmann, Nigel H. Lovell, Gary D. Housley

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Non-Viral Delivery Systems to Transport Nucleic Acids for Inherited Retinal Disorders by Md Jobair Jony, Ameya Joshi, Alekha Dash, Surabhi Shukla

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