Zou, Haoyu Tuhin, Israth Jahan Monty, Masuma Akter Luo, Shenggen Shao, Jiaqi Yan, Zhiqiang Yu, Lei
Published in
Human gene therapy
High interleukin 17A (IL-17A) expression in hepatocellular carcinoma (HCC) tissue promotes HCC development. This study explores a method to inhibit HCC growth by neutralizing IL-17A in the HCC microenvironment. A novel type 5 adenoviral vector (Ad5) that carries DNA sequences encoding specific neutralizing IL-17A recombinant antibody fragments was ...
McNally, David J. Piras, Bryan A. Willis, Catherine M. Lockey, Timothy D. Meagher, Michael M.
Published in
Molecular Therapy. Methods & Clinical Development
We have developed a simple, integrated method for cellular lysis and primary capture and recovery of AAV via hydrophobic interaction chromatography. This method results in high recoveries, substantial impurity removal, is easily scalable, and is compatible with multiple industrially relevant serotypes of AAV.
Zetsche, Bernd Abudayyeh, Omar O Gootenberg, Jonathan S Scott, David A Zhang, Feng
Published in
The Keio journal of medicine
The class 2 CRISPR-Cas endonuclease Cas12a (previously known as Cpf1) offers several advantages over Cas9, including the ability to process its own array and the requirement for just a single RNA guide. These attributes make Cas12a promising for many genome engineering applications. To further expand the suite of Cas12a tools available, we tested 1...
Chen, Joseph C Luu, Amanda R Wise, Nathan Angelis, Rolando De Agrawal, Vishal Mangini, Linley Vincelette, Jon Handyside, Britta Sterling, Harry Lo, Melanie J
...
Published in
The Journal of biological chemistry
Autosomal recessive mutations in the galactosidase β1 (GLB1) gene cause lysosomal β-gal deficiency, resulting in accumulation of galactose-containing substrates and onset of the progressive and fatal neurodegenerative lysosomal storage disease, GM1 gangliosidosis. Here, an enzyme replacement therapy (ERT) approach in fibroblasts from GM1 gangliosid...
Carrella, Sabrina Indrieri, Alessia Franco, Brunella Banfi, Sandro
Published in
Frontiers in Neuroscience
Gene therapy is proving to be an effective approach to treat or prevent ocular diseases ensuring a targeted, stable, and regulated introduction of exogenous genetic material with therapeutic action. Retinal diseases can be broadly categorized into two groups, namely monogenic and complex (multifactorial) forms. The high genetic heterogeneity of mon...
Samani, Amir A. Nalbantoglu, Josephine Brodt, Pnina
Published in
Frontiers in Oncology
Glioblastoma multiforme is an aggressive malignancy, resistant to standard treatment modalities and associated with poor prognosis. We analyzed the role of the IGF system in intracerebral glioma growth using human and rat glioma cells. The glioma cells C6 and U87MG were transduced with a genetically engineered retrovirus expressing type 1 insulin-l...
Bates, Rhiannon Huang, Wei Cao, Lei
Published in
Molecular Therapy. Methods & Clinical Development
AAV8 and a novel hybrid serotype, Rec2, show higher efficacy in transducing adipose tissue than do other serotypes. This review summarizes the advances in developing AAV vectors with enhanced adipose tropism and restricting off-target transgene expression. The challenges and strategies to develop AAV vector systems tailoring for adipose tissue are ...
Elbadawy, Hossein M. Mohammed Abdul, Mohi I. Aljuhani, Naif Vitiello, Adriana Ciccarese, Francesco Shaker, Mohamed A. Eltahir, Heba M. Palù, Giorgio Di Antonio, Veronica Ghassabian, Hanieh
...
Published in
Viruses
Despite the introduction of directly acting antivirals (DAAs), for the treatment of hepatitis C virus (HCV) infection, their cost, patient compliance, and viral resistance are still important issues to be considered. Here, we describe the generation of a novel JFH1-based HCV subgenomic replicon double reporter cell line suitable for testing differe...
Martier, Raygene Konstantinova, Pavlina
Published in
Frontiers in Neuroscience
Gene therapy is an emerging and powerful therapeutic tool to deliver functional genetic material to cells in order to correct a defective gene. During the past decades, several studies have demonstrated the potential of AAV-based gene therapies for the treatment of neurodegenerative diseases. While some clinical studies have failed to demonstrate t...
Luciani, Marco Gritti, Angela Meneghini, Vasco
Published in
Frontiers in molecular biosciences
Lysosomal storage diseases (LSDs) are a group of rare genetic conditions. The absence or deficiency of lysosomal proteins leads to excessive storage of undigested materials and drives secondary pathological mechanisms including autophagy, calcium homeostasis, ER stress, and mitochondrial abnormalities. A large number of LSDs display mild to severe ...
