Harding, Ian H. Lynch, David R. Koeppen, Arnulf H. Pandolfo, Massimo
Published in
Human Gene Therapy
Friedreich ataxia (FRDA) is an autosomal recessive inherited multisystem disease, characterized by marked differences in the vulnerability of neuronal systems. In general, the proprioceptive system appears to be affected early, while later in the disease, the dentate nucleus of the cerebellum and, to some degree, the corticospinal tracts degenerate...
Hinderer, Christian Nosratbakhsh, Brenden Katz, Nathan Wilson, James M
Published in
Human gene therapy
GM1 gangliosidosis is a rare neurodegenerative lysosomal storage disease caused by loss-of-function mutations in the gene encoding beta-galactosidase (β-gal). There are no approved treatments for GM1 gangliosidosis. Previous studies in animal models have demonstrated that adeno-associated viral (AAV) vector-mediated gene transfer to the brain can r...
Krotova, Karina Aslanidi, George
Published in
Human Gene Therapy
Adeno-associated virus (AAV)-based gene therapy is undergoing major expansion into clinical practice, with two treatments currently being granted Food and Drug Administration (FDA) approval. However, the presence of pre-existing neutralizing antibodies (NAB) is one of the significant hurdles for the clinical application of AAV vectors that signific...
Yamaguchi, Teruhide Uchida, Eriko Okada, Takashi Ozawa, Keiya Onodera, Masafumi Kume, Akihiro Shimada, Takashi Takahashi, Satoru Tani, Kenzaburo Nasu, Yasutomo
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Published in
Human Gene Therapy
The development of genome-editing technology could lead to breakthrough gene therapy. Genome editing has made it possible to easily knock out or modify a target gene, while current gene therapy using a virus vector or plasmid hampering modification with respect to gene replacement therapies. Clinical development using these genome-editing tools is ...
Krutzke, Lea Allmendinger, Ellen Hirt, Katja Kochanek, Stefan
Published in
Human Gene Therapy
Oncolytic viruses are promising anticancer agents; however, regarding their clinical efficacy, there is still significant scope for improvement. Preclinical in vivo evaluation of oncolytic viruses is mainly based on syngeneic or xenograft tumor models in mice, which is labor-intensive and time-consuming. Currently, a large proportion of development...
Shieh, Perry B. Bönnemann, Carsten G. Müller-Felber, Wolfgang Blaschek, Astrid Dowling, James J. Kuntz, Nancy L. Seferian, Andreea M.
Published in
Human Gene Therapy
Guerin, Karen Rego, Meghan Bourges, Daniela Ersing, Ina Haery, Leila Harten DeMaio, Kate Sanders, Erin Tasissa, Meron Kostman, Maya Tillgren, Michelle
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Published in
Human gene therapy
Recombinant adeno-associated virus (rAAV) vectors are increasingly popular gene delivery tools in biological systems. They are safe and lead to high-level, long-term transgene expression. rAAV are available in multiple serotypes, natural or engineered, which enable targeting to a wide array of tissues and cell types. In addition, rAAVs are relative...
Westhaus, Adrian Cabanes-Creus, Marti Rybicki, Arkadiusz Baltazar, Grober Navarro, Renina Gale Zhu, Erhua Drouyer, Matthieu Knight, Maddison Albu, Razvan F Ng, Boaz H
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Published in
Human gene therapy
Adeno-associated virus (AAV) vectors are quickly becoming the vectors of choice for therapeutic gene delivery. To date, hundreds of natural isolates and bioengineered variants have been reported. While factors such as high production titer and low immunoreactivity are important to consider, the ability to deliver the genetic payload (physical trans...
Carter, Barrie J.
Published in
Human Gene Therapy
Olbrich, Henning Theobald, Sebastian J. Slabik, Constanze Gerasch, Laura Schneider, Andreas Mach, Michael Shum, Thomas Mamonkin, Maksim Stripecke, Renata
Published in
Human Gene Therapy
Human cytomegalovirus (HCMV) reactivations are associated with lower overall survival after transplantations. Adoptive transfer of HCMV-reactive expanded or selected T cells can be applied as a compassionate use, but requires that the human leukocyte antigen-matched donor provides memory cells against HCMV. To overcome this, we developed engineered...