Results 51 to 60 of about 48,006 (264)

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

, 2004
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Braun-Falco, M., Buning, H., Hallek, M.
core   +1 more source

Cross Sectional Study of Prenatal Diagnosis Uptake Among Individuals With Genetic Conditions

Prenatal Diagnosis, EarlyView.
ABSTRACT Objective Prenatal diagnostic genetic testing allows for early identification of significant fetal conditions and enables informed decision‐making regarding management options. The aim of this study was to assess prenatal testing practice among individuals with genetic conditions.
Ebunoluwa Ojo, Roni Zemet, Allyson Nevins, Katherine Abraham, April D. Adams   +4 more
wiley   +1 more source

A Comparison between Recombinant Activated Factor VII (Aryoseven) and Novoseven in Patients with Congenital Factor VII Deficiency [PDF]

, 2015
In order to establish the efficacy and biosimilar nature of AryoSeven to NovoSeven in the treatment of congenital factor VII (FVII) deficiency, patients received either agent at 30 1/4g/kg, intravenously per week for 4 weeks, in a randomized fashion. The
Abolghasemi, H., Ahmadinejad, M., Baghaeipour, M.R., Dehdezi, B.K., Eshghi, P., Faranoush, M., Fayazfar, R., Heshmat, R., Hoorfar, H., Kamyar, K., Karimi, M., Khoeiny, B., Managhchi, M., Mehrvar, A., Mirbehbahani, N.B., Naderi, M., Toogeh, G.   +16 more
core  

Supporting Children With a Chronic Disease and Their Parents When Admitted to Hospital: A Scoping Review of Psychosocial Supports

Acta Paediatrica, EarlyView.
Importance of psychosocial support. ABSTRACT Aim The aim of this scoping review was to identify, examine, and summarize available evidence regarding psychosocial supports provided to children with a chronic disease when admitted to hospital and their parents. Method The JBI methodology for conducting and reporting scoping reviews was followed.
Lyndsay Jerusha MacKay, Nolan Lagrisolal, Una Chang, Matthew Christoffersen, K. Alix Hayden, Kathryn Birnie, Tammie Dewan   +6 more
wiley   +1 more source

Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success

EMBO Molecular Medicine, 2016
Hemophilia B is a serious hemostasis disorder due to mutations of the factor IX gene in the X chromosome. Gene therapy has gained momentum in recent years as a therapeutic option for hemophilia B.
Tuan Huy Nguyen, Ignacio Anegon
doaj   +1 more source

An assessment of the relative influence of pain coping, negative thoughts about pain, and pain acceptance on health-related quality of life among people with hemophilia [PDF]

, 2009
Many people with hemophilia are affected by chronic arthritic joint pain as well as acute bleeding pain. In this cross-sectional study, 209 men with hemophilia A or B completed the Hemophilia Pain Coping Questionnaire (HPCQ), the Chronic Pain Acceptance ...
Barakat, Baron, Barry, Choiniere, Elander, Elander, Esteve, Fischer, Fischer, Georgina Robinson, Gil, Gil, Hays, Hays, James Elander, Jensen, Jensen, John Morris, Kathryn Mitchell, Kratz, McCracken, McCracken, McCracken, McCracken, McCracken, McCracken, McCracken, McHorney, McHorney, Miners, Miners, Preacher, Roberts, Romano, Rosenstiel, Royal, Ruta, Santavirta, Solovieva, Solovieva, Stansfield, Szende, Trippoli, Turner, Turner, Vowles, Vowles, Vowles   +47 more
core   +1 more source

Therapeutic potential of small molecules that block receptor‐induced Kv7/M‐current suppression in neuroprotection, seizures, and pain

British Journal of Pharmacology, EarlyView.
Receptor‐induced Kv7/M‐current suppression increases neuronal activity and contributes to pathology of several conditions. A new class of Kv7 modulators identified in this study attenuates M‐current suppression and shows therapeutic effects. Abstract Background and Purpose Neuronal Kv7 channels generate low voltage–gated potassium currents known as the
Young Woo Kim, Pavan Kumar Behara, Lamees Alhassen, Cindy Wong, Olivier Civelli, Naoto Hoshi   +5 more
wiley   +1 more source

Strategies for Hemophilia Treatment, a literature review of current evidence

مجله دانشگاه علوم پزشکی بیرجند, 2023
Hemophilia is an inherited bleeding disorder caused by malfunctioning or lacking blood coagulation factor VIII (hemophilia A) or IX (hemophilia B). Currently, the main treatments for these X-linked diseases are replacement therapy using periodic and ...
Fahimeh Ghasemi, Nafiseh Erfanian, Samira Karbasi, Alireza Zomorodipour   +3 more
doaj  

Garadacimab for the long‐term prophylaxis of hereditary angioedema

JDDG: Journal der Deutschen Dermatologischen Gesellschaft, EarlyView.
Summary Hereditary angioedema (HAE), a rare and debilitating disease characterized by recurrent and spontaneous attacks of tissue swelling, has a high unmet therapeutic need, with many patients experiencing insufficient disease control with current prophylactic treatments.
Emel Aygören‐Pürsün, Regina Treudler, Petra Staubach, Inmaculada Martinez Saguer, Thomas Linhoff, Markus Magerl   +5 more
wiley   +1 more source

A study to determine the prevalence, clinical profile and incidence of formation of inhibitors in patients of hemophilia in north eastern part of India

Journal of Family Medicine and Primary Care, 2019
Introduction: Deficiency of factor VIII (Hemophilia A), factor IX (Hemophilia B) and Von Willebrand's factor are the most frequent coagulation defects.
Sandip Kumar, Sachidanand Sinha, Anju Bharti, Lalit P Meena, Vineeta Gupta, Jyoti Shukla   +5 more
doaj   +1 more source