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Autor:
Taku Kaitsuka, Kazuhito Tomizawa, Akihiro Shiotani, Toru Miwa, Kunio Mizutari, Takeshi Matsunobu, Eiji Yumoto, Takao Yamada, Ryosei Minoda, Farzana Hakim, Takaomi Kurioka, Hiroki Takeda, Momoko Ise
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Cell-penetrating peptides (CPPs) are short sequences of amino acids that facilitate the penetration of conjugated cargoes across mammalian cell membranes, and as such, they may provide a safe and effective method for drug delivery to the inner ear. S
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Hematopoietic stem cell (HSC) gene therapy using retroviral vectors has immense potential, but vector-mediated genotoxicity limits use in the clinic. Lentiviral vectors are less genotoxic than gammaretroviral vectors and have become the vector of cho
Autor:
Kumi Adachi, Yuko Nitahara-Kasahara, Koichi Miyake, Taku Noguchi, Takashi Okada, Atsushi Watanabe, Osamu Iijima, Sonoko Narisawa, Takashi Shimada, Hideaki Kinoshita, Shinichi Abe, José Luis Millán, Yukihiko Hirai, Noriko Miyake, Aki Nakamura-Takahashi
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Hypophosphatasia (HPP) is an inherited disease caused by genetic mutations in the gene encoding tissue-nonspecific alkaline phosphatase (TNALP). This results in defects in bone and tooth mineralization. We recently demonstrated that TNALP-deficient (
Autor:
Peter Humphries, Naomi Chadderton, Jean Bennett, Arpad Palfi, Paul F. Kenna, Jane Farrar, Kerstin Nagel-Wolfrum, Mary O'Reilly, Sophia Millington-Ward, Uwe Wolfrum
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 2, Iss, Pp-(2015)
Molecular Therapy: Methods & Clinical Development, Vol 2, Iss, Pp-(2015)
As gene therapies for various forms of retinal degeneration progress toward human clinical trial, it will be essential to have a repertoire of safe and efficient vectors for gene delivery to the target cells. Recombinant adeno-associated virus (AAV)
Autor:
Dan Goldowitz, Elizabeth M. Simpson, Simone C McInerny, William W. Hauswirth, Robert A. Holt, Stéphanie Laprise, Michelle Zhou, Elodie Portales-Casamar, Shannon E. Boye, Cletus D'Souza, Kathleen G. Banks, Sanford L. Boye, Frank M. Dyka, Wyeth W. Wasserman, Charles N. de Leeuw, Steven J.M. Jones, Magdalena I. Swanson, Lisa J. Borretta, Alice Y Chou
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 1, Iss C (2014)
Molecular Therapy: Methods & Clinical Development, Vol 1, Iss C (2014)
Critical for human gene therapy is the availability of small promoters tools to drive gene expression in a highly specific and reproducible manner. We tackled this challenge by developing human DNA MiniPromoters (MiniPs) using computational biology a