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Human Gene Therapy|August 27, 2021
Neutralizing Antibody Evasion and Transduction with Purified Extracellular Vesicle-Enveloped Adeno-Associated Virus VectorsMing Cheng, Laura Dietz, Yi Gong, et al.Human Gene Therapy|July 25, 2025
Intrastriatal Delivery of a Zinc Finger Protein Targeting the Mutant HTT Gene Allele Obviates Lipid Phenotypes in Brain and Plasma in Huntington's Disease MiceAndrew Iwanowicz, Adel Boudi, Connor Seeley, et al.Human Gene Therapy|December 3, 2025
Long-Term Follow-Up of Patients Receiving Cell and Gene Therapy ProductsLaszlo Irinyi, Barbara Mintzes, Julia Warning, et al.Human Gene Therapy|December 23, 2024
Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene TherapyPasquale Piccolo, Nicola Brunetti-PierriHuman Gene Therapy|December 26, 2024
Intracisternal AAV9-MAG-hABCD1 Vector Reverses Motor Deficits in Adult Adrenomyeloneuropathy MiceYasemin Özgür Günes, Catherine Le Stunff, Pierre BougnèresHuman Gene Therapy|February 20, 2025
AAV-Mediated Gene Transfer of WDR45 Corrects Neurological Deficits in the Mouse Model of Beta-Propeller Protein-Associated NeurodegenerationMaria Carla Carisi, Claire Shamber, Martha Bishop, et al.Human Gene Therapy|January 10, 2025
Novel Cystic Fibrosis Ferret Model Enables Visualization of CFTR Expression Cells and Genetic CFTR ReactivationFeng Yuan, Xingshen Sun, Soo Yeun Park, et al.Human Gene Therapy|August 28, 2024
Adeno-Associated Virus Vectors-a Target of Cellular and Humoral Immunity-are Expanding Their Reach Toward Hematopoietic Stem Cell Modification and ImmunotherapiesAngela E Araujo, Martin Bentler, Xabier Perez Garmendia, et al.Human Gene Therapy|August 19, 2024
Inhalation of SP-101 Followed by Inhaled Doxorubicin Results in Robust and Durable hCFTRΔR Transgene Expression in the Airways of Wild-Type and Cystic Fibrosis FerretsKatherine J D A Excoffon, Mark D Smith, Lillian Falese, et al.Human Gene Therapy|August 19, 2024
SP-101, A Novel Adeno-Associated Virus Gene Therapy for the Treatment of Cystic Fibrosis, Mediates Functional Correction of Primary Human Airway Epithelia From Donors with Cystic FibrosisKatherine J D A Excoffon, Shen Lin, Poornima Kotha Lakshmi Narayan, et al.Pageof 413