The first in-human study of chimeric antigen receptor (CAR) T cells targeting the tyrosine kinase receptor ROR1 in patients with solid tumors showed that the cells could be transferred into patients safely and expanded in vivo, according to findings from the ongoing phase I study presented at t...
In this paper, we demonstrate that the transduction of differentiated HepaRG (dHepaRG) cells can be performed successfully using lentiviral particles. The production of a functional Green Fluorescent Protein (GFP) assessed by Fluorescence Activated Cell Sorting and fluorescence microscopy is up to 16%...
Interius’s Phase 1 trial (INVISE, Injectable Vectors for In Situ Engineering) will evaluate the safety of a single INT2104 infusion in adults with refractory/relapsing B cell malignancies. The Phase 1 study is a global, two-part, multicenter, open-label, single dose design with a dose esc...
There are currently three CD19 CAR-T-cell products, i.e., Kymriah™ (lentiviral vector-based), Yescarta™ (γ-retroviral vector-based), and Tecartus™ (γ-retroviral vector-based) that have obtained marketing authorization by the FDA and EMA. All three products are autologous, gene-engin...
the phrase regarding the relevant proteins was observed by immunohistochemistry. The expression of FAM134B ended up being significantly increased in person liver disease tissue and HCC cellular lines Hep3B and Huh7. After the lentiviral vector was transfected into Hep3B cells with sh-FAM134B, resul...
The International Fanconi Anemia Gene Therapy Working Group helped the development of new generation of FA gene therapy programs, which began with a HIV-1-derived, self-inactivating lentiviral vector. RP-L102's lentiviral vector carries the FANC-A gene as part of the PGK-FANCA-WPRE expression ...
Polynucleotides 31aa-uORF of the present invention are inserted into expression vectors (i.e., a nucleic acid construct) to enable expression of the recombinant polypeptide. In one embodiment, the expression vector of the present invention includes additional sequences which render this vector suitabl...
The next generation of PSC-based clinical trials will probably include a PSC-derived dopaminergic neuron therapy for Parkinson disease, and improvements in differentiation protocols are advancing the development of other PSC-based therapies for conditions such as diabetes, liver disease and skin disorders...
Children with Hunter syndrome have a missing gene, meaning they cannot produce an important enzyme called iduronate-2-sulfatase or IDS. The gene therapy works by collecting HSCs from the patient and inserting a working copy of the gene into the HSCs using a lentiviral gene therapy vector. The ...