User profiles for J. Fakhiri

Julia Fakhiri

Scientist
Verified email at roche.com
Cited by 640

[HTML][HTML] Fantastic AAV gene therapy vectors and how to find them—random diversification, rational design and machine learning

J Becker, J Fakhiri, D Grimm - Pathogens, 2022 - mdpi.com
Parvoviruses are a diverse family of small, non-enveloped DNA viruses that infect a wide
variety of species, tissues and cell types. For over half a century, their intriguing biology and …

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

…, E Bourges, C Leborgne, N Guerchet, J Fakhiri… - Science …, 2022 - science.org
Bioengineering of viral vectors for therapeutic gene delivery is a pivotal strategy to reduce
doses, facilitate manufacturing, and improve efficacy and patient safety. Here, we engineered …

Engineered anti-CRISPR proteins for optogenetic control of CRISPR–Cas9

…, A Bietz, MC Waldhauer, K Börner, J Fakhiri… - Nature …, 2018 - nature.com
Anti-CRISPR proteins are powerful tools for CRISPR–Cas9 regulation; the ability to precisely
modulate their activity could facilitate spatiotemporally confined genome perturbations and …

[HTML][HTML] Best of most possible worlds: Hybrid gene therapy vectors based on parvoviruses and heterologous viruses

J Fakhiri, D Grimm - Molecular Therapy, 2021 - cell.com
Parvoviruses and especially the adeno-associated virus (AAV) species provide an exciting
and versatile platform for the rational design or molecular evolution of human gene-therapy …

Cell-specific CRISPR–Cas9 activation by microRNA-dependent expression of anti-CRISPR proteins

…, S Grosse, K Rapti, C Domenger, J Fakhiri… - Nucleic acids …, 2019 - academic.oup.com
The rapid development of CRISPR–Cas technologies brought a personalized and targeted
treatment of genetic disorders into closer reach. To render CRISPR-based therapies precise …

Relevance of assembly-activating protein for adeno-associated virus vector production and capsid protein stability in mammalian and insect cells

…, AK Herrmann, K Börner, J Fakhiri… - Journal of …, 2017 - Am Soc Microbiol
The discovery that adeno-associated virus 2 (AAV2) encodes an eighth protein, called
assembly-activating protein (AAP), transformed our understanding of wild-type AAV biology. …

[HTML][HTML] Pre-arrayed pan-AAV peptide display libraries for rapid single-round screening

…, J Weinmann, A Sacher, P Bayer, C Stüllein, J Fakhiri… - Molecular therapy, 2020 - cell.com
Display of short peptides on the surface of adeno-associated viruses (AAVs) is a powerful
technology for the generation of gene therapy vectors with altered cell specificities and/or …

[HTML][HTML] Novel chimeric gene therapy vectors based on adeno-associated virus and four different mammalian bocaviruses

J Fakhiri, MA Schneider, J Puschhof, M Stanifer… - … Therapy-Methods & …, 2019 - cell.com
Parvoviruses are highly attractive templates for the engineering of safe, efficient, and
specific gene therapy vectors, as best exemplified by adeno-associated virus (AAV). Another …

Breaking the sound barrier: Towards next-generation AAV vectors for gene therapy of hearing disorders

J Fakhiri, LD Landegger, D Grimm - Hearing Research, 2022 - Elsevier
Owing to the advances in transgenic animal technology and the advent of the next-generation
sequencing era, over 120 genes causing hereditary hearing loss have been identified by …

[PDF][PDF] Ex vivo/in vivo gene editing in hepatocytes using “all-in-one” CRISPR-adeno-associated virus vectors with a self-linearizing repair template

SA Krooss, Z Dai, F Schmidt, A Rovai, J Fakhiri… - Iscience, 2020 - cell.com
Adeno-associated virus (AAV)-based vectors are considered efficient and safe gene
delivery systems in gene therapy. We combined two guide RNA genes, Cas9, and a self-linearizing …