Cluster of Excellence CellNetworks,
Department of Infectious Diseases,
Virology,
Cluster of Excellence CellNetworks, Department of Infectious Diseases, Virology
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Primary human cells differ in their susceptibility to rAAV-2-mediated gene transfer and duration of reporter gene expression.
Journal of virological methods Sep, 2002 | Pubmed ID: 12270659
Production methods for gene transfer vectors based on adeno-associated virus serotypes.
Methods (San Diego, Calif.) Oct, 2002 | Pubmed ID: 12413413
Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6.
Molecular therapy : the journal of the American Society of Gene Therapy Jun, 2003 | Pubmed ID: 12788658
Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy.
Blood Oct, 2003 | Pubmed ID: 12791653
Adeno-associated virus vectors for short hairpin RNA expression.
Methods in enzymology , 2005 | Pubmed ID: 15644194
Increased maintenance and persistence of transgenes by excision of expression cassettes from plasmid sequences in vivo.
Human gene therapy May, 2005 | Pubmed ID: 15916481
Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype.
Journal of virology Jan, 2006 | Pubmed ID: 16352567
Improved cardiac gene transfer by transcriptional and transductional targeting of adeno-associated viral vectors.
Cardiovascular research Apr, 2006 | Pubmed ID: 16448634
Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways.
Nature May, 2006 | Pubmed ID: 16724069
The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9.
Journal of virology Oct, 2006 | Pubmed ID: 16973587
A two-hybrid screen identifies cathepsins B and L as uncoating factors for adeno-associated virus 2 and 8.
Molecular therapy : the journal of the American Society of Gene Therapy Feb, 2007 | Pubmed ID: 17235311
Combinatorial RNAi: a winning strategy for the race against evolving targets?
Molecular therapy : the journal of the American Society of Gene Therapy May, 2007 | Pubmed ID: 17311009
Rapid and stable knockdown of an endogenous gene in retinal pigment epithelium.
Human gene therapy Oct, 2007 | Pubmed ID: 17892416
RNAi and gene therapy: a mutual attraction.
Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program , 2007 | Pubmed ID: 18024667
Therapeutic application of RNAi: is mRNA targeting finally ready for prime time?
The Journal of clinical investigation Dec, 2007 | Pubmed ID: 18060021
Hepatic parenchymal replacement in mice by transplanted allogeneic hepatocytes is facilitated by bone marrow transplantation and mediated by CD4 cells.
Hepatology (Baltimore, Md.) Feb, 2008 | Pubmed ID: 18220289
In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses.
Journal of virology Jun, 2008 | Pubmed ID: 18400866
Silencing of hepatic fatty acid transporter protein 5 in vivo reverses diet-induced non-alcoholic fatty liver disease and improves hyperglycemia.
The Journal of biological chemistry Aug, 2008 | Pubmed ID: 18524776
Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic.
Molecular therapy : the journal of the American Society of Gene Therapy Sep, 2008 | Pubmed ID: 18665161
Small silencing RNAs: state-of-the-art.
Advanced drug delivery reviews Jul, 2009 | Pubmed ID: 19427885
Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice.
Molecular therapy : the journal of the American Society of Gene Therapy Jan, 2010 | Pubmed ID: 19844192
Six RNA viruses and forty-one hosts: viral small RNAs and modulation of small RNA repertoires in vertebrate and invertebrate systems.
PLoS pathogens Feb, 2010 | Pubmed ID: 20169186
Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver.
The Journal of clinical investigation Sep, 2010 | Pubmed ID: 20697157
Thermodynamic stability of small hairpin RNAs highly influences the loading process of different mammalian Argonautes.
Proceedings of the National Academy of Sciences of the United States of America May, 2011 | Pubmed ID: 21576459
Cellular RNA interference mechanisms. Preface.
Progress in molecular biology and translational science , 2011 | Pubmed ID: 21846566
When cellular networks run out of control: global dysregulation of the RNAi machinery in human pathology and therapy.
Progress in molecular biology and translational science , 2011 | Pubmed ID: 21846572
To go, or not to go, that is the question - six personal reflections on how geographic mobility may affect your career and life.
BioEssays : news and reviews in molecular, cellular and developmental biology Oct, 2011 | Pubmed ID: 21858845
The dose can make the poison: lessons learned from adverse in vivo toxicities caused by RNAi overexpression.
Silence , 2011 | Pubmed ID: 22029761
Fate tracing of mature hepatocytes in mouse liver homeostasis and regeneration.
The Journal of clinical investigation Dec, 2011 | Pubmed ID: 22105172
Expression determinants of mammalian argonaute proteins in mediating gene silencing.
Nucleic acids research Dec, 2011 | Pubmed ID: 22210886
AAV8-mediated in vivo overexpression of miR-155 enhances the protective capacity of genetically attenuated malarial parasites.
Molecular therapy : the journal of the American Society of Gene Therapy Dec, 2014 | Pubmed ID: 25189739
Zinc finger nuclease-based double-strand breaks attenuate malaria parasites and reveal rare microhomology-mediated end joining.
Genome biology Nov, 2015 | Pubmed ID: 26573820
Death receptor-based enrichment of Cas9-expressing cells.
BMC biotechnology Feb, 2016 | Pubmed ID: 26883910
Blocking sense-strand activity improves potency, safety and specificity of anti-hepatitis B virus short hairpin RNA.
EMBO molecular medicine 09, 2016 | Pubmed ID: 27473329
TALEN/CRISPR-mediated engineering of a promoterless anti-viral RNAi hairpin into an endogenous miRNA locus.
Nucleic acids research 01, 2017 | Pubmed ID: 27614072
Plasmodium berghei EXP-1 interacts with host Apolipoprotein H during Plasmodium liver-stage development.
Proceedings of the National Academy of Sciences of the United States of America Feb, 2017 | Pubmed ID: 28137845
Next-generation AAV vectors for clinical use: an ever-accelerating race.
Virus genes Oct, 2017 | Pubmed ID: 28762205
Relevance of Assembly-Activating Protein for Adeno-associated Virus Vector Production and Capsid Protein Stability in Mammalian and Insect Cells.
Journal of virology 10, 2017 | Pubmed ID: 28768875
Small But Increasingly Mighty: Latest Advances in AAV Vector Research, Design, and Evolution.
Human gene therapy 11, 2017 | Pubmed ID: 28835125
Split Cas9, Not Hairs - Advancing the Therapeutic Index of CRISPR Technology.
Biotechnology journal Sep, 2018 | Pubmed ID: 29316283
Regulation of Adult CNS Axonal Regeneration by the Post-transcriptional Regulator Cpeb1.
Frontiers in molecular neuroscience , 2017 | Pubmed ID: 29379413
High-Throughput Dissection of AAV-Host Interactions: The Fast and the Curious.
Journal of molecular biology 08, 2018 | Pubmed ID: 29782834
Impact of the Assembly-Activating Protein on Molecular Evolution of Synthetic Adeno-Associated Virus Capsids.
Human gene therapy 01, 2019 | Pubmed ID: 29978729
AAVvector-mediated in vivo reprogramming into pluripotency.
Nature communications 07, 2018 | Pubmed ID: 29985406
Engineered anti-CRISPR proteins for optogenetic control of CRISPR-Cas9.
Nature methods 11, 2018 | Pubmed ID: 30377362
A Robust and All-Inclusive Pipeline for Shuffling of Adeno-Associated Viruses.
ACS synthetic biology 01, 2019 | Pubmed ID: 30513195
Novel Chimeric Gene Therapy Vectors Based on Adeno-Associated Virus and Four Different Mammalian Bocaviruses.
Molecular therapy. Methods & clinical development Mar, 2019 | Pubmed ID: 30766894
Rapid and Simple Screening of CRISPR Guide RNAs (gRNAs) in Cultured Cells Using Adeno-Associated Viral (AAV) Vectors.
Methods in molecular biology (Clifton, N.J.) , 2019 | Pubmed ID: 30912043
Cell-specific CRISPR-Cas9 activation by microRNA-dependent expression of anti-CRISPR proteins.
Nucleic acids research 07, 2019 | Pubmed ID: 30982889
Severe Human Bocavirus 1 Respiratory Tract Infection in an Immunodeficient Child With Fatal Outcome.
The Pediatric infectious disease journal 09, 2019 | Pubmed ID: 31033910
Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors.
Gene therapy 04, 2020 | Pubmed ID: 31624368
Ex Vivo/In vivo Gene Editing in Hepatocytes Using "All-in-One" CRISPR-Adeno-Associated Virus Vectors with a Self-Linearizing Repair Template.
iScience Jan, 2020 | Pubmed ID: 31887661
Pre-arrayed Pan-AAV Peptide Display Libraries for Rapid Single-Round Screening.
Molecular therapy : the journal of the American Society of Gene Therapy 04, 2020 | Pubmed ID: 32105604
Impact of Natural or Synthetic Singletons in the Capsid of Human Bocavirus 1 on Particle Infectivity and Immunoreactivity.
Journal of virology 05, 2020 | Pubmed ID: 32213611
Computational design of anti-CRISPR proteins with improved inhibition potency.
Nature chemical biology 07, 2020 | Pubmed ID: 32284602
Lab-Scale Production of Recombinant Adeno-Associated Viruses (AAV) for Expression of Optogenetic Elements.
Methods in molecular biology (Clifton, N.J.) , 2020 | Pubmed ID: 32651911
Production, Processing, and Characterization of Synthetic AAV Gene Therapy Vectors.
Biotechnology journal Jan, 2021 | Pubmed ID: 32975881
Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants.
Nature communications 10, 2020 | Pubmed ID: 33116134
Breaking the sound barrier: Towards next-generation AAV vectors for gene therapy of hearing disorders.
Hearing research 01, 2022 | Pubmed ID: 33268240
Optogenetic control of Neisseria meningitidis Cas9 genome editing using an engineered, light-switchable anti-CRISPR protein.
Nucleic acids research 03, 2021 | Pubmed ID: 33330940
'Hit and run' therapy averts macular degeneration.
Nature biomedical engineering 02, 2021 | Pubmed ID: 33580229
A universal protocol for isolating retinal ON bipolar cells across species via fluorescence-activated cell sorting.
Molecular therapy. Methods & clinical development Mar, 2021 | Pubmed ID: 33665228
Characterization of the GBoV1 Capsid and Its Antibody Interactions.
Viruses 02, 2021 | Pubmed ID: 33672786
Best of most possible worlds: Hybrid gene therapy vectors based on parvoviruses and heterologous viruses.
Molecular therapy : the journal of the American Society of Gene Therapy 12, 2021 | Pubmed ID: 33831556
A Versatile In Vivo System to Study Myc in Cell Reprogramming.
Methods in molecular biology (Clifton, N.J.) , 2021 | Pubmed ID: 34019296
Membrane-destabilizing ionizable phospholipids: Novel components for organ-selective mRNA delivery and CRISPR-Cas gene editing.
Signal transduction and targeted therapy 05, 2021 | Pubmed ID: 34035211
The angiopoietin-Tie2 pathway regulates Purkinje cell dendritic morphogenesis in a cell-autonomous manner.
Cell reports 08, 2021 | Pubmed ID: 34407407
Neutralizing Antibody Evasion and Transduction with Purified Extracellular Vesicle-Enveloped Adeno-Associated Virus Vectors.
Human gene therapy 12, 2021 | Pubmed ID: 34445894
An RNA Interference/Adeno-Associated Virus Vector-Based Combinatorial Gene Therapy Approach Against Hepatitis E Virus.
Hepatology communications 04, 2022 | Pubmed ID: 34719133
Lentiviral and adeno-associated vectors efficiently transduce mouse T lymphocytes when targeted to murine CD8.
Molecular therapy. Methods & clinical development Dec, 2021 | Pubmed ID: 34729380
Adeno-Associated Viruses (AAV) and Host Immunity - A Race Between the Hare and the Hedgehog.
Frontiers in immunology , 2021 | Pubmed ID: 34777364
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models.
Nature communications 11, 2021 | Pubmed ID: 34819506
Ex vivo and in vivo suppression of SARS-CoV-2 with combinatorial AAV/RNAi expression vectors.
Molecular therapy : the journal of the American Society of Gene Therapy 05, 2022 | Pubmed ID: 35038579
Natural killer cells act as an extrinsic barrier for in vivo reprogramming.
Development (Cambridge, England) 04, 2022 | Pubmed ID: 35420133
Right on target: The next class of efficient, safe, and specific RNAi triggers.
Molecular therapy. Nucleic acids Jun, 2022 | Pubmed ID: 35505965
Identification of adeno-associated virus variants for gene transfer into human neural cell types by parallel capsid screening.
Scientific reports 05, 2022 | Pubmed ID: 35589936
Intranasal application of adeno-associated viruses: a systematic review.
Translational research : the journal of laboratory and clinical medicine Oct, 2022 | Pubmed ID: 35597541
Fantastic AAV Gene Therapy Vectors and How to Find Them-Random Diversification, Rational Design and Machine Learning.
Pathogens (Basel, Switzerland) Jul, 2022 | Pubmed ID: 35890005
Boosters for adeno-associated virus vector (AAV) (r)evolution.
Cytotherapy Aug, 2022 | Pubmed ID: 35999132
In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice.
Nature communications 09, 2022 | Pubmed ID: 36064805
Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders.
Science advances 09, 2022 | Pubmed ID: 36129972
Determination of AAV properties by single amino acids: Go(o)d is in the details.
Molecular therapy. Methods & clinical development Dec, 2022 | Pubmed ID: 36212908
Kleopatra Rapti1,2,
Olena Maiakovska1,2,
Jonas Becker1,2,
Joanna Szumska1,2,
Margarita Zayas1,2,
Felix Bubeck1,2,
Jixin Liu1,2,
Emma Gerstmann1,2,
Chiara Krämer1,2,
Ellen Wiedtke1,2,
Dirk Grimm1,2,3,4,5
1Department of Infectious Diseases/Virology, Section Viral Vector Technologies, Medical Faculty, University of Heidelberg,
2BioQuant Center, BQ0030, University of Heidelberg,
3, Cluster of Excellence CellNetworks,
4, German Center for Infection Research (DZIF),
5, German Center for Cardiovascular Research (DZHK)
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