Results 51 to 60 of about 1,008,568 (216)

GENE THERAPY IN MUCOPOLYSACCHARIDOSIS TYPE IIIA: CASE REPORTS

Slovenska pediatrija, 2022
Mucopolysaccharidoses are a group of rare lysosomal storage diseases. The clinical signs develop gradually, the impairment is progressive and multiple organs are affected. With the currently known treatment options, the patient cannot be cured.
Benjamin Lah, Tadej Jalšovec, Ana Drole Torkar, Jana Kodrič, Saba Battelino, Mojca Žerjav Tanšek, Tadej Battelino, Urh Grošelj   +7 more
doaj   +1 more source

Chromosome Transplantation: A Possible Approach to Treat Human X-linked Disorders

Molecular Therapy: Methods & Clinical Development, 2020
Many human genetic diseases are associated with gross mutations such as aneuploidies, deletions, duplications, or inversions. For these “structural” disorders, conventional gene therapy, based on viral vectors and/or on programmable nuclease-mediated ...
Marianna Paulis, Lucia Susani, Alessandra Castelli, Teruhiko Suzuki, Takahiko Hara, Letizia Straniero, Stefano Duga, Dario Strina, Stefano Mantero, Elena Caldana, Lucia Sergi Sergi, Anna Villa, Paolo Vezzoni   +12 more
doaj   +1 more source

Mesenchymal and neural stem cell differentiation into Purkinje-like cells: Morphological study

Journal of Taibah University Medical Sciences
الملخص: أهداف البحث: تُعدّ خلايا بيركنجي فئةً محورية من الخلايا العصبية المُثبِّطة في الجهاز العصبي المركزي، وقد ارتبطخلل وظيفتها بطيفٍ من الاضطرابات العصبية.
Maeda H. Mohammad, PhD, Ahmed M. Al-Shammari, PhD, Ahmad A. Aljuboori, PhD, Rafal Hussamildeen, PhD, Zaid A. Al-Hilli, PhD   +4 more
doaj   +1 more source

2-Deoxyglucose and Newcastle Disease Virus Synergize to Kill Breast Cancer Cells by Inhibition of Glycolysis Pathway Through Glyceraldehyde3-Phosphate Downregulation

Frontiers in Molecular Biosciences, 2019
Targeting cancer cells metabolism is promising strategy in inhibiting cancer cells progression that are known to exhibit increased aerobic glycolysis. We used the glucose analog 2-Deoxyglucose (2-DG) as a competitor molecule of glucose.
Ahmed Majeed Al-Shammari, Amer Hasan Abdullah, Zainab Majid Allami, Nahi Y. Yaseen   +3 more
doaj   +1 more source

Maturity onset diabetes of the young (mody): the importance of combined ngs and mlpa genetic testing [PDF]

, 2019
Maturity onset diabetes of the young (MODY) is a rare form of diabetes characterized by an onset of hyperglycaemia before 25 years of age, autosomal dominant inheritance and in some cases insulin independence.
Zdravković, Vera, Anđelković, Marina, Ugrin, Milena, Ješić, Maja, Pavlović, Sonja, Šajić, Silvija, Komazec, Jovana   +6 more
core  

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

Neural Excitatory/Inhibitory Imbalance in Motor Aging: From Genetic Mechanisms to Therapeutic Challenges

Biology
Neural excitatory/inhibitory (E/I) imbalance plays a pivotal role in the aging process. However, despite its significant impact, the role of E/I imbalance in motor dysfunction and neurodegenerative diseases has not received sufficient attention.
Xuhui Chen, Ya Wang, Yongning Zhang, Xucheng Li, Le Zhang, Shangbang Gao, Cuntai Zhang   +6 more
doaj   +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

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

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