Results 41 to 50 of about 1,008,568 (216)

Splicing modulation therapy in the treatment of genetic diseases [PDF]

, 2014
Virginia Arechavala-Gomeza,1 Bernard Khoo,2 Annemieke Aartsma-Rus3 1Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Bizkaia, Spain; 2Endocrinology, Division of Medicine, University College London, London, UK; 3Department ...
Khoo B, Aartsma-Rus A, Arechavala-Gomeza V   +2 more
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Targeted therapy of acute myeloid leukemia [PDF]

, 2015
Advances in the understanding of the genetic underpinnings of acute myeloid leukemia are rapidly being translated into novel treatment strategies.
Jessica K Altman, Ronan Swords, Carneiro, B.A., Francis J Giles, Altman, Jessica K, Platanias, Leonidas C, Platanias, L.C., Gert Ossenkoppele, Kaplan, Jason B, Kaplan, J.B., Leonidas C Platanias, Jason B Kaplan, Swords, R., Benedito A Carneiro, Swords, Ronan, Altman, J.K., Carneiro, Benedito A, Giles, Francis J, Ossenkoppele, Gert, Ossenkoppele, G., Giles, F.J.   +20 more
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Advances in cell transplantation therapy and gene therapy in Parkinson's disease

Chinese Journal of Contemporary Neurology and Neurosurgery, 2022
The role of cell transplantation therapy and gene therapy in the treatment of Parkinson's disease (PD) has attracted more and more attention. It has developed from laboratory research tools to clinical products for patients.
XU Bao⁃lei, CHAN Piu
doaj   +1 more source

HLA‐Bw4 homozygosity is associated with an impaired CD4 T cell recovery after initiation of antiretroviral therapy [PDF]

, 2008
We assessed the influence of human leukocyte antigen (HLA) alleles HLA-Bw4 and HLA-Bw6 on CD4 T cell recovery after starting successful combination antiretroviral therapy in 265 individuals. The median gains in the CD4 T cell count after 4 years were 258
Battegay, Manuel, Günthard, Huldrych F, Gaudieri, S., Swiss, HIV Cohort Study, John, Mina, Nolan, David, James, Ian, Furrer, H, Schmid, P, Battegay, M., McKinnon, E, Rauch, A., Swiss HIV Cohort Study, ., McKinnon, E., Telenti, A, Günthard, Huldrych F., Schmid, Patrick, Rauch, A, John, M., James, I, Furrer, Hansjakob, Hirschel, Bernard, Telenti, Amalio, Schmid, P., Furrer, H., Rauch, Andri, John, M, Günthard, H.F., Mallal, Simon, Telenti, A., McKinnon, Elizabeth, Nolan, D., Günthard, H F, Hirschel, B, Günthard, H. F., Battegay, M, Hirschel, B., Nolan, D, Mallal, S, James, I., Gaudieri, Silvana, Mallal, S., Gaudieri, S   +42 more
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Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model

Nature Communications
Adeno-associated virus (AAV)-mediated gene silencing offers a promising strategy for achieving durable therapeutic effects with a single administration. Mutations in the human superoxide dismutase 1 (hSOD1) gene, inherited in an autosomal dominant manner,
Fang Wan, Jinchen He, Hong Ma, Debora PiresFerreira, Veena Kumanan, Ji Sun Lee, Xiupeng Chen, Ran He, Qin Su, Thomas L. Gallagher, Sha Zhu, Gabriela Toro Cabrera, Lingzhi Zhao, Joan Shen, Alisha Gruntman, Robert H. Brown, Zuoshang Xu, Guangping Gao, Jun Xie   +18 more
doaj   +1 more source

Optogenetics as a Novel Therapeutic Approach for Ocular Disease

Journal of Clinical & Translational Ophthalmology
Optogenetics is a field that emerged with the goal of studying the physiology of nerve cells by selectively expressing opsins—channel proteins that can be activated by light exposure.
Enzo Maria Vingolo, Simona Mascolo, Mattia Calabro, Filippo Miccichè, Mirko Barresi   +4 more
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Optimizing milk-derived exosome carriers through systematic cationic material screening for efficient pulmonary-targeted mRNA delivery

陆军军医大学学报
Objective To construct an engineered milk-derived exosome (mExos) vector for efficient messenger RNA (mRNA) delivery to the respiratory tract by systematically screening cationic modification materials.
LUO Mingxing, LIAO Rui
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AON-based degradation of c.151C>T mutant COCH transcripts associated with dominantly inherited hearing impairment DFNA9

Molecular Therapy: Nucleic Acids, 2021
The c.151C>T founder mutation in COCH is a frequent cause of late-onset, dominantly inherited hearing impairment and vestibular dysfunction (DFNA9) in the Dutch/Belgian population.
Erik de Vrieze, Jorge Cañas Martín, Jolien Peijnenborg, Aniek Martens, Jaap Oostrik, Simone van den Heuvel, Kornelia Neveling, Ronald Pennings, Hannie Kremer, Erwin van Wijk   +9 more
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Personalizing HIV therapy, mission impossible? [PDF]

, 2013
Sustained HIV suppression depends on a number of factors including therapy adherence, management of side effects, viral resistance and individual characteristics of patients and therapeutic settings.
Hentig, Nils von
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Novel genetic alterations and their impact on target therapy response in head and neck squamous cell carcinoma [PDF]

, 2019
Xiaohua Jiang,* Jing Ye,* Zhihuai Dong, Sunhong Hu, Mang Xiao Department of Otolaryngology Head and Neck Surgery, Sir Run Shaw Hospital, College of Medicine, Zhejiang University, Hangzhou, Zhejiang, China *These authors contributed equally to this work ...
Hu S, Xiao M, Jiang X, Dong Z, Ye J
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