Neuromuscular diseases: genomics-driven advances by Anna Cho

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Cardiac Gene Therapy With Phosphodiesterase 2A Limits Remodeling and Arrhythmias in Mouse Models of Heart Failure by Rima Kamel, Aurélia Bourcier, Jean Piero Margaria, Valentin Jin, Audrey Varin, Agnès Hivonnait, Françoise Mercier‐Nomé, Delphine Mika, Alessandra Ghigo, Flavien Charpentier, Vincent Algalarrondo, Emilio Hirsch, Rodolphe Fischmeister, Grégoire Vandecasteele, Jérôme Leroy

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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) a... 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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Editorial: Gene therapy and genome editing for metabolic liver disorders by Fanny Collaud, Fanny Collaud, Giulia Bortolussi

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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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Advances in Nucleic Acid Drugs and Gene Therapies based on Animal Models of Duchenne Muscular Dystrophy by LIU Siyu, LAI Yuezhao, GUO Wenting, CHEN Xuejin

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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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Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations by Lucas Tricoli, Sunetra Sase, Julia L. Hacker, Vi Pham, Maxwell Chappell, Laura Breda, Stephanie N. Hurwitz, Naoto Tanaka, Carlo Castruccio Castracani, Amaliris Guerra, Zhongqi Hou, Lars Schlotawa, Karthikeyan Radhakrishnan, Matthew Hogenauer, Aoife Roche, John Everett, Frederic Bushman, Peter Kurre, Rebecca Ahrens-Nicklas, Laura A. Adang, Adeline L. Vanderver, Stefano Rivella

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Clinical trials to gene therapy development and production in Brazil: a review by Jonas Alex Morales Saute, Virginia Picanço-Castro, Ana Carolina de Freitas Lopes, João Batista da Silva Júnior, Johanna Henriques Nehm, Franciele dos Santos Maciel, Ida Vanessa Doederlein Schwartz, Juliana Maria Ferraz Sallum, Margareth Castro Ozelo, Roberto Giugliani, Martin Bonamino, Hilda Petrs-Silva, Guilherme Baldo

Subjects: “…Gene therapy products…”
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Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies by Prathibha Babu Chandraprabha, Manoj Kumar K. Azhagiri, Vigneshwaran Venkatesan, Wendy Magis, Kirti Prasad, Sevanthy Suresh, Aswin Anand Pai, Srujan Marepally, Alok Srivastava, Kumarasamypet Murugesan Mohankumar, David I. K. Martin, Saravanabhavan Thangavel

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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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Advances in Gene Therapy for Neurologic Disorders: An Overview by Pallavi Chand, K. Trideva Sastri, Ashish Singh Chauhan, Souvik Chakraborty, Vikash Jakhmola

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<i>Ex Vivo</i> Regional Gene Therapy Compared to Recombinant BMP-2 for the Treatment of Critical-Size Bone Defects: An In Vivo Single-Cell RNA-Sequencing Study by Arijita Sarkar, Matthew C. Gallo, Jennifer A. Bell, Cory K. Mayfield, Jacob R. Ball, Mina Ayad, Elizabeth Lechtholz-Zey, Stephanie W. Chang, Osamu Sugiyama, Denis Evseenko, Jay R. Lieberman

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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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Advancements in the Treatment of Mucopolysaccharidoses: From Established Therapies to Gene Therapy by Joanna Wanat, Wojciech Homa, Aleksandra Warunek, Gabriela Gronowicz, Izabela Dzikowska, Agata Siejka, Daria Stefaniak, Weronika Zielińska, Michał Chról

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The role of hospital pharmacists in supporting the appropriate and safe use of CGT/ATMPs: a scoping review of current insights by Junnan Shi, Xianwen Chen, Hao Hu, Carolina Oi Lam Ung

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