Results 61 to 70 of about 36,968 (256)

CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich's Ataxia. [PDF]

, 2020
Friedreich's ataxia (FRDA) is an autosomal recessive neurodegenerative disorder caused by expansion of GAA repeats in intron 1 of the frataxin (FXN) gene, leading to significant decreased expression of frataxin, a mitochondrial iron-binding protein.
Cherqui, Stephanie, Haquang, Joseph H, Luebeck, Jens, Mali, Prashant, Rainaldi, Joseph N, Rocca, Celine J, Sharma, Jay, Shi, Yanmeng   +7 more
core  

Chimeric Antigen Receptor T‐Cells in Myasthenia Gravis: Advances, Safety Challenges, and Future Directions

Muscle &Nerve, EarlyView.
ABSTRACT This review examines the emerging application of chimeric antigen receptor (CAR) T‐cell therapy in myasthenia gravis (MG), with emphasis on safety, efficacy signals, and future therapeutic potential in treatment‐refractory disease. A comprehensive literature search was conducted across PubMed, medRxiv, bioRxiv, and Google Scholar for studies ...
Tobias Hegelmaier, Alexander Duscha, Christiane Desel, Sarah Stassen, Tim Krzemien, Ann‐Katrin Hennemann, Vaia Pappa, Aiden Haghikia   +7 more
wiley   +1 more source

Systematic Review of Low‐Density Lipoprotein Cholesterol Apheresis for the Treatment of Familial Hypercholesterolemia

Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease, 2016
BackgroundApheresis is an important treatment for reducing low‐density lipoprotein cholesterol (LDL‐C) in patients with familial hypercholesterolemia (FH). We systematically reviewed the current literature surrounding LDL‐C apheresis for FH.
Anthony Wang, Akshara Richhariya, Shravanthi R. Gandra, Brian Calimlim, Lisa Kim, Ruben G. W. Quek, Robert J. Nordyke, Peter P. Toth   +7 more
doaj   +1 more source

Comparison of early mortality between leukapheresis and non-leukapheresis in adult acute myeloid leukemia patients with hyperleukocytosis: a systematic review and meta-analysis

Hematology, 2022
Objectives One of the treatment modalities that can be used for hyperleukocytosis is leukapheresis. However, the result of studies showing the benefit of early mortality through the use of leukapheresis versus no leukapheresis is still inconclusive ...
Ikhwan Rinaldi, Noorwati Sutandyo, Kevin Winston   +2 more
doaj   +1 more source

Quality of Life in Patients With Chronic Inflammatory Demyelinating Polyneuropathy

Muscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare disorder of the nervous system that detrimentally influences health‐related quality of life (HRQoL). Evidence regarding factors influencing HRQoL in CIDP is scarce.
Tobias C. Blum, Hendrik van der Wurp, David Mengel, Linda Fraune, Norbert Sommer, Mark Stettner, Björn Tackenberg, Richard Dodel   +7 more
wiley   +1 more source

Acute effects of low density lipoprotein apheresis on metabolic parameters of apolipoprotein B

Journal of Lipid Research, 2000
Apheresis is a treatment option for patients with severe hypercholesterolemia and coronary artery disease. It is unknown whether such therapy changes kinetic parameters of lipoprotein metabolism, such as apolipoprotein B (apoB) secretion rates ...
Klaus G. Parhofer, P. Hugh R. Barrett, Thomas Demant, Peter Schwandt   +3 more
doaj   +1 more source

Bortezomib-containing multimodality treatment for antibody-mediated rejection with anti-HLA and anti-AT1R antibodies after kidney transplantation [PDF]

, 2017
For decades, the human leukocyte antigen (HLA) complex has been considered the primary target of antibody-mediated rejection (AMR), and treatment strategies have mainly focused on anti-HLA antibodies.
Favi, Evaldo, Iesari, Samuele, Lai, Quirino, Pisani, Francesco   +3 more
core   +1 more source

When Technology Falters: Ensuring Donor Safety and System Reliability Amid Apheresis Technical Challenges

Artificial Organs, EarlyView.
This study reviews three adverse events during single donor platelet collection using root cause analysis. Causes included human error, equipment defects, and procedural lapses. Though all donors fully recovered, the incidents underscore the need for strict SOP adherence, staff training, and donor safety to maintain trust and donation retention.
Bhagyashri Surve, Chitra Dewanji, Aditi Khanna, Suryatapa Saha, Shashank Ojha   +4 more
wiley   +1 more source

Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management. A position paper from the Consensus Panel on Familial Hypercholesterolaemia of the European Atherosclerosis Society [PDF]

, 2014
AIMS: Homozygous familial hypercholesterolaemia (HoFH) is a rare life-threatening condition characterized by markedly elevated circulating levels of low-density lipoprotein cholesterol (LDL-C) and accelerated, premature atherosclerotic cardiovascular ...
Averna, M, AVERNA, Maurizio, Boileau, C, Boileau, C, Boren, J, Boren, J, Bruckert, E, Bruckert, E, Catapano, A, Catapano, A, Chapman, M, Chapman, M, Chapman, M, Cuchel, M, Cuchel, M, Defesche, J, Defesche, J, Descamps, O, Descamps, O, Ginsberg, H, Ginsberg, H, Ginsberg, H, Hegele, R, Hegele, R, Hovingh, G, Hovingh, G, Humphries, S, Humphries, S, Kovanen, P, Kovanen, P, Kuivenhoven, J, Kuivenhoven, J, Masana, L, Masana, L, Nordestgaard, B, Nordestgaard, B, Pajukanta, P, Pajukanta, P, Parhofer, K, Parhofer, K, Raal, F, Raal, F, Ray, K, Ray, K, Santos, R, Santos, R, Stalenhoef, A, Stalenhoef, A, Steinhagen Thiessen, E, Steinhagen Thiessen, E, Stroes, E, Stroes, E, Taskinen, M, Taskinen, M, Tybjaerg Hansen, A, Tybjaerg Hansen, A, Watts, G, Watts, G, Wiegman, A, Wiegman, A, Wiklund, O, Wiklund, O.   +61 more
core   +1 more source

G‐CSF for Mobilizing CD34+ Cells in Individuals With SCD: A Word of Caution

American Journal of Hematology, EarlyView.
Akshay Sharma, Allistair Abraham, Monica Bhatia, María Cancio, Sonali Chaudhury, Selim Corbacioglu, Gregory M. T. Guilcher, Ashish O. Gupta, Rabi Hanna, Matthew M. Heeney, Tami John, Lakshmanan Krishnamurti, James L. LaBelle, Hemalatha G. Rangarajan, Shalini Shenoy, Elizabeth Stenger, Crawford Strunk, Courtney D. Fitzhugh   +17 more
wiley   +1 more source