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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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

Subjects: “…Gene therapy…”
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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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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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The Diagnosis and Treatment of Branch Retinal Vein Occlusions: An Update by Diana-Maria Darabuş, Rodica Georgiana Dărăbuş, Mihnea Munteanu

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Current views on etiology, diagnosis, epidemiology and gene therapy of maturity onset diabetes in the young by Lilya U. Dzhemileva, Elena N. Zakharova, Anna O. Goncharenko, Maria V. Vorontsova, S. A. Rumyantsev, Natalia G. Mokrysheva, Marina Y. Loguinova, Vladimir P. Chekhonin

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Endothelial and neuronal engagement by AAV-BR1 gene therapy alleviates neurological symptoms and lipid deposition in a mouse model of Niemann-Pick type C2 by Charlotte Laurfelt Munch Rasmussen, Signe Frost Frederiksen, Christian Würtz Heegaard, Maj Schneider Thomsen, Eva Hede, Bartosz Laczek, Jakob Körbelin, Daniel Wüstner, Louiza Bohn Thomsen, Markus Schwaninger, Ole N. Jensen, Torben Moos, Annette Burkhart

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