Brunetti-Pierri, Nicola Grove, Nathan C Zuo, Yu Edwards, Rachel Palmer, Donna Cerullo, Vincenzo Teruya, Jun Ng, Philip
Published in
Human gene therapy
Although the desire to develop gene therapy for hemophilia B is high, safety remains a concern. Therefore, improving the therapeutic index of gene therapy vectors is an important goal. Thus, we evaluated the use of three bioengineered factor IX (FIX) variants with improved catalytic activity in the context of the helper-dependent adenoviral vector....
Eberling, Jamie L Kells, Adrian P Pivirotto, Philip Beyer, Janine Bringas, John Federoff, Howard J Forsayeth, John Bankiewicz, Krystof S
Published in
Human gene therapy
We investigated the safety and neuroregenerative potential of an adeno-associated virus (AAV2) containing human glial cell line-derived neurotrophic factor (GDNF) in an MPTP primate model of Parkinson's disease. Dopaminergic function was evaluated by positron emission tomography with 6-[(18)F]fluoro-l-m-tyrosine (FMT) before and after AAV2-GDNF or ...
Chiu, Yahui Grace Bowers, William J Lim, Seung T Ryan, Deborah A Federoff, Howard J
Published in
Human gene therapy
The herpes simplex virus (HSV)-based amplicon is a versatile vaccine platform that has been preclinically vetted as a gene-based immunotherapeutic for cancer, HIV, and neurodegenerative disorders. Although it is well known that injection of dendritic cells (DCs) transduced ex vivo with helper virus-free HSV amplicon vectors expressing disease-relev...
Huang, Xiaopei Yang, Yiping
Published in
Human gene therapy
Recombinant viral vectors such as adenovirus and adenovirus-associated virus have been used widely as vehicles for gene therapy applications because of the high efficiency with which they transfer genes into a wide spectrum of cells in vivo. However, enthusiasm for the use of viral vectors in gene therapy has been tempered by significant problems o...
Chuang, Chi-Mu Monie, Archana Wu, Annie Mao, Chih-Ping Hung, Chien-Fu
Published in
Human gene therapy
There is an urgent need for innovative therapies against ovarian cancer, one of the leading causes of death from gynecological cancers in the United States. Immunotherapy employing Toll-like receptor (TLR) ligands, such as CpG oligodeoxynucleotides (CpG-ODN), may serve as a potentially promising approach in the control of ovarian tumors. The CpG-OD...
Song, Yuhu Lou, Howard H Boyer, Julie L Limberis, Maria P Vandenberghe, Luk H Hackett, Neil R Leopold, Philip L Wilson, James M Crystal, Ronald G
Published in
Human gene therapy
Cystic fibrosis is characterized by deficiency of the cystic fibrosis transmembrane conductance regulator (CFTR), a Cl(-) transporter. The packaging constraints of adeno-associated viral (AAV) vectors preclude delivery of both an active promoter and CFTR cDNA to target cells. We hypothesized that segmental trans-splicing, in which two AAV vectors d...
Park, Paul J Colletti, Evan Ozturk, Ferhat Wood, Josh A Tellez, Joe Almeida-Porada, Graça Porada, Christopher
Published in
Human gene therapy
The possibility of permanent genetic changes to the germline is central to the bioethics of in utero gene therapy (IUGT) because of the concern of inadvertent potentially deleterious alterations to the gene pool. Despite presumed protection of the male germline due to early germ cell (GC) compartmentalization, we reported that GCs within the develo...
van den Berg, Joost H Nujien, Bastiaan Beijnen, Jos H Vincent, Andrew van Tinteren, Harm Kluge, Jörn Woerdeman, Leonie A E Hennink, Wim E Storm, Gert Schumacher, Ton N
...
Published in
Human gene therapy
The intradermal administration of DNA vaccines by tattooing is a promising delivery technique for genetic immunization, with proven high immunogenicity in mice and in nonhuman primates. However, the parameters that result in optimal expression of DNA vaccines that are applied by this strategy to human skin are currently unknown. To address this iss...
Barraza, Román A Rasmussen, Carol A Loewen, Nils Cameron, J Douglas Gabelt, B'Ann T Teo, Wu-Lin Kaufman, Paul L Poeschla, Eric M
Published in
Human gene therapy
We injected lentiviral vectors into the eyes of live nonhuman primates to assess potential for glaucoma gene therapy. Anterior chambers of five cynomolgus monkeys were injected with green fluorescent protein (GFP)-encoding feline immunodeficiency viral vectors. The monkeys were monitored for in vivo transgene expression and clinical parameters. The...
Wehling, Peter Reinecke, Julio Baltzer, Axel W A Granrath, Markus Schulitz, Klaus P Schultz, Carl Krauspe, Rüdiger Whiteside, Theresa W Elder, Elaine Ghivizzani, Steven C
...
Published in
Human gene therapy
This paper provides the first evidence of a clinical response to gene therapy in human arthritis. Two subjects with rheumatoid arthritis received ex vivo, intraarticular delivery of human interleukin-1 receptor antagonist (IL-1Ra) cDNA. To achieve this, autologous synovial fibroblasts were transduced with a retrovirus, MFG-IRAP, carrying IL-1Ra as ...
