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Author Details

Leszek Lisowski
Military Institute of Medicine
2004
67
25
Prashant Mali (CM4AI)
PMIDPaper TitleJournal TitlePublished Year
35976053Adeno-associated virus serotype 2 capsid variants for improved liver-directed gene therapy.Hepatology2023
38081924AAV capsid bioengineering in primary human retina models.Sci Rep2023
37553343Long-term ex situ normothermic perfusion of human split livers for more than 1 week.Nat Commun2023
37651221Direct optogenetic activation of upper airway muscles in an acute model of upper airway hypotonia mimicking sleep onset.Sleep2023
37674904Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes.Mol Ther Methods Clin Dev2023
37451880In Search of Adeno-Associated Virus Vectors With Enhanced Cardiac Tropism for Gene Therapy.Heart Lung Circ2023
36858919Liver splitting during normothermic machine perfusion: a novel method to combine the advantages of both in-situ and ex-vivo techniques.HPB (Oxford)2023
36786126Molecular mechanisms of amyotrophic lateral sclerosis as broad therapeutic targets for gene therapy applications utilizing adeno-associated viral vectors.Med Res Rev2023
36700121Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution.Mol Ther Methods Clin Dev2023
35297686AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression.Hum Gene Ther2022
36544150The future of recombinant host defense peptides.Microb Cell Fact2022
36472367Proteome-wide systems genetics identifies UFMylation as a regulator of skeletal muscle function.Elife2022
36369267Optogenetic restoration of high sensitivity vision with bReaChES, a red-shifted channelrhodopsin.Sci Rep2022
35867177Liver-specific deletion of miR-181ab1 reduces liver tumour progression via upregulation of CBX7.Cell Mol Life Sci2022
36382885A human adenovirus encoding IFN-γ can transduce Tasmanian devil facial tumour cells and upregulate MHC-I.J Gen Virol2022
35882232Phosphoproteomics of three exercise modalities identifies canonical signaling and C18ORF25 as an AMPK substrate regulating skeletal muscle function.Cell Metab2022
35881862Gene Editing and Rett Syndrome: Does It Make the Cut?CRISPR J2022
32935224A drug-tunable Flt23k gene therapy for controlled intervention in retinal neovascularization.Angiogenesis2021
33916893Neurological Disorders Associated with WWOX Germline Mutations-A Comprehensive Overview.Cells2021
34977275Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8.Mol Ther Methods Clin Dev2021
34988473Potential Applications for Targeted Gene Therapy to Protect Against Anthracycline Cardiotoxicity: <i>JACC: CardioOncology</i> Primer.JACC CardioOncol2021
34703837Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys.Mol Ther Methods Clin Dev2021
34428180The self-peptide repertoire plays a critical role in transplant tolerance induction.J Clin Invest2021
34485808The intersection of vector biology, gene therapy, and hemophilia.Res Pract Thromb Haemost2021
34095344Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids.Mol Ther Methods Clin Dev2021
33316848Selection of a novel AAV2/TNFAIP3 vector for local suppression of islet xenograft inflammation.Xenotransplantation2021
33197448The Balance of Stromal BMP Signaling Mediated by GREM1 and ISLR Drives Colorectal Carcinogenesis.Gastroenterology2021
31511858Systemic AAV8-mediated delivery of a functional copy of muscle glycogen phosphorylase (Pygm) ameliorates disease in a murine model of McArdle disease.Hum Mol Genet2020
32000541High-Throughput <i>In Vitro</i>, <i>Ex Vivo,</i> and <i>In Vivo</i> Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction.Hum Gene Ther2020
32603653In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation.Mol Ther2020
32908003Restoring the natural tropism of AAV2 vectors for human liver.Sci Transl Med2020
32978399Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing.Nat Commun2020
32490035Attenuation of Heparan Sulfate Proteoglycan Binding Enhances <i>In Vivo</i> Transduction of Human Primary Hepatocytes with AAV2.Mol Ther Methods Clin Dev2020
30027761Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort.Hum Gene Ther2019
31752513A User's Guide to the Inverted Terminal Repeats of Adeno-Associated Virus.Hum Gene Ther Methods2019
32039406Efficient <i>in vivo</i> editing of OTC-deficient patient-derived primary human hepatocytes.JHEP Rep2019
31649959Targeting Adeno-Associated Virus Vectors for Local Delivery to Fractures and Systemic Delivery to the Skeleton.Mol Ther Methods Clin Dev2019
30218651Optogenetic approaches to vision restoration.Exp Eye Res2019
29055620Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.Mol Ther2018
30220458An Atypical Parvovirus Drives Chronic Tubulointerstitial Nephropathy and Kidney Fibrosis.Cell2018
30101152Bioengineered Viral Platform for Intramuscular Passive Vaccine Delivery to Human Skeletal Muscle.Mol Ther Methods Clin Dev2018
30534580Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality.Mol Ther Methods Clin Dev2018
30274736Directed Evolution of Adeno-Associated Virus Vectors in Human Cardiomyocytes for Cardiac Gene Therapy.Heart Lung Circ2018
29754775In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation.Mol Ther2018
28628105Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome.Nat Genet2017
29233960Multiplexed in vivo homology-directed repair and tumor barcoding enables parallel quantification of Kras variant oncogenicity.Nat Commun2017
27064447RNA interference-induced hepatotoxicity results from loss of the first synthesized isoform of microRNA-122 in mice.Nat Med2016
26291407Adeno-associated virus serotypes for gene therapeutics.Curr Opin Pharmacol2015
24390279Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant.Mol Ther2014
25070322Second generation codon optimized minicircle (CoMiC) for nonviral reprogramming of human adult fibroblasts.Methods Mol Biol2014
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Collaborators

Ana M Moreno
University of California San Diego
Co-authored papers 2
Shijun Hu
Co-authored papers 2
Shyni Varghese
Duke University School of Medicine
Co-authored papers 2
Fyodor D Urnov
Innovative Genomics Institute, University of California berkeley
Co-authored papers 2
Kang Zhang
College of Animal Science and Technology, Guangxi University
Co-authored papers 2
Hiroyuki Nakai
Oregon Health and Science University
Co-authored papers 2
Jasmine Tat
University of California, USA Amgen Inc.
Co-authored papers 2
Dhruva Katrekar
University of California San Diego
Co-authored papers 2
Alex W Hewitt
Menzies Institute for Medical Research, University of Tasmania
Co-authored papers 2
Prashant Mali (CM4AI)
University of California San Diego
Co-authored papers 2
John Naughton
Salk Institute for Biological Studies
Co-authored papers 2
Yu-Ru V Shih
Duke University
Co-authored papers 2
Jie Zhu
Guangzhou Women and Children's Medical Center, Guangzhou Medical University
Co-authored papers 2
Jessica Cabotaje
University of California
Co-authored papers 2
John Marlett
Salk Institute for Biological Studies
Co-authored papers 2
Xin Fu
Xin Hua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
Co-authored papers 2
Michael Ott
Co-authored papers 1
Alistair R R Forrest
RIKEN Center for Integrative Medical Sciences
Co-authored papers 1
Monte M Winslow
Stanford University School of Medicine
Co-authored papers 1
Kimberly Gilmour
NIHR Great Ormond Street Hospital BRC
Co-authored papers 1
Jose G Vilches-Moure
Co-authored papers 1
Anthony W Purcell
Monash Biomedicine Discovery Institute, Monash University
Co-authored papers 1
Daniel L Worthley
South Australian Health and Medical Research Institute
Co-authored papers 1
Anthony E Oro
Stanford University
Co-authored papers 1
Karen Reue
Co-authored papers 1
Susan L Woods
Adelaide Medical School, University of Adelaide
Co-authored papers 1
Mark C Gillies
The University of Sydney Save Sight Institute
Co-authored papers 1
Tamsin R M Lannagan
Adelaide Medical School, University of Adelaide
Co-authored papers 1
Matthew J Watt
Co-authored papers 1
Nigel G Laing
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