Results 61 to 70 of about 1,003,750 (315)

Pupillometric analysis for assessment of gene therapy in Leber Congenital Amaurosis patients [PDF]

, 2012
Background: Objective techniques to assess the amelioration of vision in patients with impaired visual function are needed to standardize efficacy assessment in gene therapy trials for ocular diseases.
Melillo Paolo, Jean Bennett, Francesca Simonelli, Simonelli Francesca, BENNETT J, Testa, Francesco, Francesco Testa, Paolo Melillo, Leandro Pecchia, Rossi, Settimio, Pecchia Leandro, Testa Francesco, Bennett, Jean, M.D., Pecchia, Leandro, PECCHIA L, Melillo, Paolo, Settimio Rossi, Simonelli, Francesca, Bennett Jean, Rossi Settimio   +19 more
core   +1 more source

Efficacy and Safety Analysis of Roxarestat in Regulating Renal Anemia in Patients on Maintenance Hemodialysis

Therapeutic Apheresis and Dialysis, EarlyView.
ABSTRACT Objective To compare the efficacy and safety of roxarestat versus recombinant human erythropoietin (rhEPO) in the management of renal anemia in patients undergoing maintenance hemodialysis. Methods This was a prospective, open‐label, randomized controlled trial.
Lingling Chen, Junjie Zhu, Qiaonan Ge
wiley   +1 more source

Genetic and Genomic Landscape of Secondary and Therapy-Related Acute Myeloid Leukemia

, 2020
A subset of acute myeloid leukemia (AML) arises either from an antecedent myeloid malignancy (secondary AML, sAML) or as a complication of DNA-damaging therapy for other cancers (therapy-related myeloid neoplasm, t-MN).
Alexandra Higgins, Mithun Vinod Shah
core   +1 more source

Hereditary Transthyretin-Related Amyloidosis: Genetic Heterogeneity and Early Personalized Gene Therapy

, 2022
Point mutations of the transthyretin (TTR) gene are related with hereditary amyloidosis (hATTR). The number of people affected by this rare disease is only partially estimated.
Sturiale, Valentina, Bruno, Francesca, Concetta Federico, Francesca Bruno, Brancato, Desiree, Valentina Sturiale, Ketty Dugo, Salvatore Saccone, Desiree Brancato, Saccone, Salvatore, Dugo, Ketty, Federico, Concetta   +11 more
core   +1 more source

Infection Control Practices for Vascular Access Management in Hemodialysis: Results From a Nationwide Survey of Japanese National University Hospitals

Therapeutic Apheresis and Dialysis, EarlyView.
ABSTRACT Introduction Bloodstream infections due to repeated vascular access (VA) puncture and circuit connections remain major concerns in hemodialysis. Therefore, we examined current practices for glove, disinfectant, and personal protective equipment (PPE) use according to VA type in national university hospitals in Japan.
Aiko Yamada, Makoto Harada, Tadahiko Kikugawa, Takeshi Nakata, Koji Hashimoto, Tsuneo Konta, Yuji Kamijo   +6 more
wiley   +1 more source

GMP-manufactured CRISPR/Cas9 technology as an advantageous tool to support cancer immunotherapy

Journal of Experimental & Clinical Cancer Research
Background CRISPR/Cas9 system to treat human-related diseases has achieved significant results and, even if its potential application in cancer research is improving, the application of this approach in clinical practice is still a nascent technology ...
M Caforio, S Iacovelli, C Quintarelli, F Locatelli, Valentina Folgiero   +4 more
doaj   +1 more source

A genetic validation study reveals a role of vitamin D metabolism in the response to interferon-alfa-based therapy of chronic hepatitis C [PDF]

, 2012
Background: To perform a comprehensive study on the relationship between vitamin D metabolism and the response to interferon-α-based therapy of chronic hepatitis C.
Jörg Bojunga, Raffaele Malinverni, Gerlach, Tilman J., Gerlach, Tilman J, Tilman J Gerlach, Swiss Hepatitis C Cohort Study Group,, Berg, T., Kutalik, Z., Stephanie Bibert, Christian M Lange, Geier, Andreas, Dufour, Jean-François Jacques, Thomas Berg, Malinverni, Raffaele, Regenass, S., Burgisser, Philippe, Regenass, Stephan, Badenhoop, Klaus, Bojunga, Jörg, Lange, Christian M, Dufour, J. F., Stephan Regenass, Gerlach, Tilman Joern, Swiss Hepatitis C Cohort Study Group, Bochud, P. Y., Dufour, Jean-Francois, Bojunga, J., Andreas Geier, Stefan Zeuzem, Negro, F., Cerny, A., Zoltan Kutalik, Lange, Christian, Pierre-Yves Bochud, Sarrazin, C., Tobias Müller, Sarrazin, Christoph, Lange, C. M., Cerny, Andreas, Kutalik, Zoltán, Bibert, S., Malinverni, R., Moradpour, Darius, Christoph Sarrazin, Burgisser, P., Gerlach, T. J., Moradpour, D., Bibert, Stephanie, Klaus Badenhoop, Zeuzem, S., Negro, Francesco, Müller, Tobias, Badenhoop, K., Andreas Cerny, Bochud, Pierre-Yves, Geier, A., Heim, M. H., Muller, T., Francesco Negro, Berg, Thomas, Markus H Heim, Heim, Markus H., Darius Moradpour, Jean-Francois Dufour, Philippe Burgisser, Heim, Markus H, Zeuzem, Stefan   +66 more
core   +1 more source

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

, 2022
We are in an emerging era of gene-based therapeutics with significant promise for rare genetic disorders. The potential is particularly significant for genetic central nervous system disorders that have begun to achieve Food and Drug Administration ...
Tauscher-Wisniewski, Sitra, Bönnemann, Carsten, Wang, Dan, Zoghbi, Huda Y, Buxbaum, Joseph D, Kleiman, Robin J, Davidson, Beverly L, Bradbury, Allison M, Shaywitz, Adam J, Sahin, Mustafa, Tifft, Cynthia J, Gray, Steven J, Gray-Edwards, Heather L, Gao, Guangping, Berry-Kravis, Elizabeth, Flotte, Terence R, Corcoran, Gavin R   +16 more
core   +1 more source

Genetic therapy for pain management

Current Review of Pain, 2000
It has been suggested that there are two main approaches to the use of gene therapy in the treatment of chronic disease, which has been confirmed by recent studies in animals.
S P, Wilson, D C, Yeomans
openaire   +3 more sources

Therapeutic Apheresis in Nigeria: A Multi‐Center Summary of Abstracts From the Inaugural Nigerian Society for Apheresis Scientific Meeting

Therapeutic Apheresis and Dialysis, EarlyView.
ABSTRACT Background Therapeutic apheresis (TA) is an established treatment modality for hematologic, neurologic, and immunologic disorders, yet access remains severely limited in sub‐Saharan Africa. Donor apheresis, including platelet apheresis collection from healthy donors, represents an important complementary modality supporting blood product ...
Nosa Bazuaye, Chimezie Okwuonu, Evelyn Idam, Folasade Adelekan‐Popoola, Shakirat Gold‐Olufadi, Olufunto Olufela Kalejaiye, Abdullateef Ogala, Zinnah Aloziem, Blossom Amamchukwu, Patience Elimia, Mkpoikanabasi Obot‐Obot, Henry Mbakwe, Praises Maduagwuna, Olatunji Akinrodoye, Chiamaka Anyanwu, Benjamin Anyanwu, Adedamola Akinsiku, Oluwatoyin A. Akinsiku, Muzamil O. Hassan, Fatiu A. Arogundade, Olalekan E. Ojo, Homayemem Weli, Jesupelumi Adenihun, Aderemi Adelaja, Ebun Bamgboye, Olalekan Olatise, Ade E. Faponle, Ayoola Odeyemi, Michael Muoka, Stephen Asaolu, Qudrat Busari, Blessing Dawudu, Michael Naiyeju, Rasheed A. Balogun   +33 more
wiley   +1 more source