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Journal of Neurosurgery. Pediatrics|August 15, 2025
Robot-assisted intrathalamic infusion for gene therapy in young children: surgical considerationsRrita Daci, Mohammed Salman Shazeeb, Brittany Owusu-Adjei, et al.
Biorxiv : the Preprint Server for Biology|July 14, 2025
A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse ModelsJenny Gao, Nathan Bamidele, Debora Pires-Ferreira, et al.
Quarterly Journal of Experimental Psychology (2006)|May 26, 2017
Learning from where 'eye' remotely look or point: Impact on number line estimation error in adultsThomas Gallagher-Mitchell, Victoria Simms, Damien Litchfield
Radiology. Cardiothoracic Imaging|July 12, 2021
Accelerated in Vivo Cardiac Diffusion-Tensor MRI Using Residual Deep Learning-based Denoising in Participants with ObesityKellie Phipps, Maaike van de Boomen, Robert Eder, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 11, 2003
Successful transgene expression with serial doses of aerosolized rAAV2 vectors in rhesus macaquesAnne C Fischer, Suzanne E Beck, Carolina I Smith, et al.
Human Gene Therapy|April 30, 2013
Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correctionLi Zhong, Nirav Malani, Mengxin Li, et al.
Human Gene Therapy|March 31, 2018
In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT DeficiencyChun-Qing Song, Dan Wang, Tingting Jiang, et al.
The Journal of Biological Chemistry|September 1, 2005
The short apical membrane half-life of rescued {Delta}F508-cystic fibrosis transmembrane conductance regulator (CFTR) results from accelerated endocytosis of {Delta}F508-CFTR in polarized human airway epithelial cellsAgnieszka Swiatecka-Urban, Andrea Brown, Sophie Moreau-Marquis, et al.
Human Gene Therapy|June 6, 2018
Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal DystrophyMark E Pennesi, Richard G Weleber, Paul Yang, et al.
Molecular Therapy. Methods & Clinical Development|March 6, 2024
Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaquesMeghan Blackwood, Alisha M Gruntman, Qiushi Tang, et al.
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