Results 21 to 30 of about 1,003,750 (315)

DNA Edition: ad portas of a revolution in genetic manipulation

Iatreia, 2020
In biological sciences, genetic therapy constitutes a “trend topic” since its beginning. Development of new technologies in bioengineering as zinc-finger nucleases (ZFN), Transcription activator-like effector nu-cleases (TALEN) and ...
Madariaga Perpiñán, Ithzayana , Duque Restrepo, Juan Camilo, Ayala Ramírez, Paola, García Robles, Reggie   +3 more
doaj   +1 more source

The First Homozygote Mutation c.499G>T (Asp167Tyr) in the RPE65 Gene Encoding Retinoid Isomerohydrolase Causing Retinal Dystrophy

Current Issues in Molecular Biology, 2022
RPE65, an abundant membrane-associated protein present in the retinal pigment epithelium (RPE), is a vital retinoid isomerase necessary for regenerating 11-cis-retinaldehyde from all-trans retinol in the visual cycle.
Mirjana Bjeloš, Ana Ćurić, Benedict Rak, Mladen Bušić, Biljana Kuzmanović Elabjer   +4 more
doaj   +1 more source

Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles

, 2022
Fibrodysplasia ossificans progressiva (FOP) is a rare and devastating genetic disease, in which soft connective tissue is converted into heterotopic bone through an endochondral ossification process.
Shim, Jae-Hyuck, Delai, Patricia L R, Madhuri, Vrisha, De Cunto, Carmen L, de Ruiter, Ruben D, Schoenmaker, Ton, Zasloff, Michael, Friedman, Clive S, Gao, Guangping, Cho, Tae-Joon, Morhart, Rolf, Grunwald, Zvi, di Rocco, Maja, Haga, Nobuhiko, de Vries, Teun J, Hsiao, Edward C, Pals, Gerard, Netelenbos, Coen, Diecidue, Robert J, Kaplan, Frederick, Smilde, Bernard J, Scott, Christiaan, Eekhoff, Elisabeth M W, Pignolo, Robert J, Micha, Dimitra   +24 more
core   +1 more source

In Vitro Synergistic Enhancement of Newcastle Disease Virus to 5-Fluorouracil Cytotoxicity against Tumor Cells

Biomedicines, 2016
Background: Chemotherapy is one of the antitumor therapies used worldwide in spite of its serious side effects and unsatisfactory results. Many attempts have been made to increase its activity and reduce its toxicity.
Ahmed M. Al-Shammari, Marwa I. Salman, Yahya D. Saihood, Nahi Y. Yaseen, Khansaa Raed, Hiba Kareem Shaker, Aesar Ahmed, Aseel Khalid, Ahlam Duiach   +8 more
doaj   +1 more source

The seroprevalence of neutralizing antibodies against the adeno-associated virus capsids in Japanese hemophiliacs

Molecular Therapy: Methods & Clinical Development, 2022
Adeno-associated virus (AAV) vectors are promising modalities of gene therapy to address unmet medical needs. However, anti-AAV neutralizing antibodies (NAbs) hamper the vector-mediated therapeutic effect.
Yuji Kashiwakura, Nemekhbayar Baatartsogt, Shoji Yamazaki, Azusa Nagao, Kagehiro Amano, Nobuaki Suzuki, Tadashi Matsushita, Akihiro Sawada, Satoshi Higasa, Naoya Yamasaki, Teruhisa Fujii, Taemi Ogura, Hideyuki Takedani, Masashi Taki, Takeshi Matsumoto, Jun Yamanouchi, Michio Sakai, Masako Nishikawa, Yutaka Yatomi, Koji Yada, Keiji Nogami, Ryota Watano, Takafumi Hiramoto, Morisada Hayakawa, Nobuhiko Kamoshita, Akihiro Kume, Hiroaki Mizukami, Shizukiyo Ishikawa, Yoichi Sakata, Tsukasa Ohmori   +29 more
doaj   +1 more source

Genetic analyses reveal a role for vitamin D insufficiency in HCV-associated hepatocellular carcinoma development [PDF]

, 2013
Background: Vitamin D insufficiency has been associated with the occurrence of various types of cancer, but causal relationships remain elusive. We therefore aimed to determine the relationship between genetic determinants of vitamin D serum levels and ...
Jörg Bojunga, Stéphanie Bibert (3279381), Lange, Christian M., Mullhaupt, B., Ochi Hidenori, Thomas Berg (186372), Müller Tobias, Ochi, H., David Semela (416437), Gouttenoire, J., Müllhaupt, Beat, Kenichi Morikawa (416434), Nischalke, Hans-Dieter, Ulrich Spengler (205545), Ochi, Hidenori, Lange, Christian M, Dufour, J. F., David Semela, Malinverni Raffaele, Meri Gorgievski-Hrisoho (416435), Bochud Pierre-Yves, Francesco Negro (205592), Semela, David, Beat Müllhaupt (416436), Nischalke, H. D., Chayama, Kazuaki, Bochud, P. Y., Gouttenoire Jérôme, Negro, F., Cerny, A., Zoltan Kutalik, Kazuaki Chayama (128675), Lange, Christian, Negro Francesco, Pierre-Yves Bochud, Morikawa, Kenichi, Bibert, S., Malinverni, R., Spengler, U., Bibert, Stéphanie, Moradpour, Darius, Darius Moradpour (15815), Bochud, P.Y., Dufour, J.F., Hans-Dieter Nischalke (416433), Heim Markus H., Raffaele Malinverni (316140), Jean-François Dufour, Meri Gorgievski-Hrisoho, Berg Thomas, Miki, D., Vinciguerra, Manlio, Hidenori Ochi (128649), Gorgievski-Hrisoho, M., Andreas Cerny, Dufour, Jean-François, Kazuaki Chayama, Ulrich Spengler, Heim, M. H., Muller, T., Hans-Dieter Nischalke, Francesco Negro, Gorgievski-Hrisoho, Meri, Berg, Thomas, Markus H Heim, Darius Moradpour, Chayama, K., Kenichi Morikawa, Miki Daiki, Dufour Jean-François, Pierre-Yves Bochud (205597), Daiki Miki (128628), Miki, Daiki, Raffaele Malinverni, Bojunga Jörg, Jérôme Gouttenoire, Spengler Ulrich, Berg, T., Nischalke, H.D., Hidenori Ochi, Spengler, Ulrich, Cerny Andreas, Kutalik, Z., Zoltan Kutalik (3965672), Andreas Cerny (189787), Christian M Lange, Gouttenoire, Jérôme, Dufour, Jean-François Jacques, Gorgievski, Meri, Thomas Berg, Malinverni, Raffaele, Moradpour Darius, Beat Müllhaupt, Semela David, Daiki Miki, Chayama Kazuaki, Bojunga, Jörg, Stéphanie Bibert, Gorgievski-Hrisoho Meri, Tobias Müller (163591), Swiss Hepatitis C Cohort Study Group, Christian M. Lange (316130), Bojunga, J., Kutalik Zoltan, Tobias Müller, Lange, C. M., Lange Christian M., Cerny, Andreas, Markus H. Heim (237987), Lange, C.M., Kutalik, Zoltán, Heim, M.H., Nischalke Hans-Dieter, Moradpour, D., Morikawa, K., Semela, D., Jörg Bojunga (316147), Morikawa Kenichi, Negro, Francesco, Müller, Tobias, Hiroshima Liver Study Group, Bochud, Pierre-Yves, Bibert Stéphanie, Heim, Markus H., Müllhaupt Beat, Heim, Markus H, Jérôme Gouttenoire (235184), Jean-François Dufour (5654965)   +127 more
core   +1 more source

Genetic therapies for neurological disorders [PDF]

Human Genetics, 2021
AbstractIn recent years, it has become increasingly apparent that many neurological disorders are underpinned by a genetic aetiology. This has resulted in considerable efforts to develop therapeutic strategies which can treat the disease-causing mutation, either by supplying a functional copy of the mutated gene or editing the genomic sequence. In this
Laura FitzPatrick, Adrian Bird
openaire   +3 more sources

Durability of transgene expression after rAAV gene therapy

, 2022
Recombinant adeno-associated virus (rAAV) gene therapy has the potential to transform the lives of patients with certain genetic disorders by increasing or restoring function to affected tissues.
Schulz, Martin, Levy, Daniel I, McCarty, Douglas, Gao, Guangping, Muhuri, Manish   +4 more
core   +1 more source

Genetic Basis and Therapies for Vascular Anomalies [PDF]

Circulation Research, 2021
Vascular and lymphatic malformations represent a challenge for clinicians. The identification of inherited and somatic mutations in important signaling pathways, including the PI3K (phosphoinositide 3-kinase)/AKT (protein kinase B)/mTOR (mammalian target of rapamycin), RAS (rat sarcoma)/RAF (rapidly accelerated fibrosarcoma)/MEK (mitogen-activated ...
Angela Queisser, Emmanuel Seront, Laurence M. Boon, Miikka Vikkula   +3 more
openaire   +3 more sources

Translating the combination of gene therapy and tissue engineering for treating recessive dystrophic epidermolysis bullosa [PDF]

European Cells & Materials, 2018
The combination of gene therapy and tissue engineering is one of the most promising strategies for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
A Dakiw Piaceski, D Larouche, K Ghani, F Bisson, S Cortez Ghio, S Larochelle, VJ Moulin, M Caruso, L Germain   +8 more
doaj   +1 more source