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Lista de obras de Leszek Lisowski

A User's Guide to the Inverted Terminal Repeats (ITR) of Adeno-Associated Virus

scientific article published on 21 November 2019

AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence.

artículo científico publicado en 2012

Adeno-associated virus serotypes for gene therapeutics.

artículo científico

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort

artículo científico publicado en 2018

An Atypical Parvovirus Drives Chronic Tubulointerstitial Nephropathy and Kidney Fibrosis

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.

artículo científico publicado en 2017

Bioengineered Viral Platform for Intramuscular Passive Vaccine Delivery to Human Skeletal Muscle

artículo científico publicado en 2018

Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality

scientific article published on 01 November 2018

Current status of globin gene therapy for the treatment of beta-thalassaemia.

artículo científico publicado en 2008

Directed Evolution of Adeno-Associated Virus Vectors in Human Cardiomyocytes for Cardiac Gene Therapy

article

Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes

scientific article published on 27 December 2019

Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing

scientific article published on 25 September 2020

Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B mice

artículo científico publicado en 2008

Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging.

artículo científico publicado en 2012

Genome editing of isogenic human induced pluripotent stem cells recapitulates long QT phenotype for drug testing

artículo científico publicado en 2014

High-Throughput In Vitro, Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction

artículo científico publicado en 2020

Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa

artículo científico publicado en 2014

Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome.

artículo científico publicado en 2017

In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation.

artículo científico publicado en 2018

Locus control region elements HS1 and HS4 enhance the therapeutic efficacy of globin gene transfer in beta-thalassemic mice.

artículo científico publicado en 2007

Multiplexed in vivo homology-directed repair and tumor barcoding enables parallel quantification of Kras variant oncogenicity. ⬇️

artículo científico publicado en 2017

Optogenetic approaches to vision restoration

scientific article published on 13 September 2018

Progress toward the genetic treatment of the beta-thalassemias.

artículo científico publicado en 2005

RNA interference-induced hepatotoxicity results from loss of the first synthesized isoform of microRNA-122 in mice.

artículo científico publicado en 2016

Restoring the natural tropism of AAV2 vectors for human liver

scientific article published on 01 September 2020

Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression.

artículo científico publicado en 2012

Second generation codon optimized minicircle (CoMiC) for nonviral reprogramming of human adult fibroblasts.

artículo científico publicado en 2014

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

artículo científico publicado en 2013

Selection of a novel AAV2/TNFAIP3 vector for local suppression of islet xenograft inflammation

scientific article published on 14 December 2020

Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant.

artículo científico publicado en 2014

Systemic AAV8-mediated delivery of a functional copy of muscle glycogen phosphorylase (Pygm) ameliorates disease in a murine model of McArdle disease

artículo científico publicado en 2020

Targeting Adeno-Associated Virus Vectors for Local Delivery to Fractures and Systemic Delivery to the Skeleton

artículo científico publicado en 2019

The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA.

artículo científico publicado en 2013

The balance of stromal BMP signaling mediated by GREM1 and ISLR drives colorectal carcinogenesis

artículo científico publicado en 2020

Transient in vivo beta-globin production after lentiviral gene transfer to hematopoietic stem cells in the nonhuman primate

artículo científico publicado en 2009

rAAV-mediated tumorigenesis: still unresolved after an AAV assault

artículo científico publicado en 2012

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