Results 61 to 70 of about 12,392,515 (266)

Specific functional knee phenotypes are associated with increased osteoarthritis progression: A ≥10‐year longitudinal study

Knee Surgery, Sports Traumatology, Arthroscopy, EarlyView.
Abstract Purpose To evaluate osteoarthritis (OA) progression across functional knee phenotypes (FKPs) over a follow‐up period of ≥10 years. Methods This retrospective longitudinal study included 388 patients (764 knees) with healthy knees or early OA (Kellgren–Lawrence grade ≤1) at baseline and a minimum follow‐up of 10 years.
Sang Jun Song, Hyo Beom Lee, Cheol Hee Park   +2 more
wiley   +1 more source

Hemophilia a genetic disorder: diagnosis, treatment and prognosis [PDF]

, 2016
Hemophilia a genetic disorder with patient’s inability to stop bleeding. There are two main types of hemophilia, hemophiliaA due to not enough clotting factor VIII and hemophilia B due to not enough factor IX,and acquired hemophilia A(AHA) caused by ...
AM Sharifa, Murtaza Mustafa, Ell. Illzam, Firdaus Mohd Hayati, Nornazirah Azizan, Ay Moktar   +5 more
core   +2 more sources

POLYMORPHISM OF THROMBOPHILIA GENES AND THEIR ROLE IN DEVELOPMENT OF DIFFERENT DISEASE PHENOTYPES AND THROMBOTIC COMPLICATIONS IN HEMOPHILIA PATIENTS

Сибирский научный медицинский журнал, 2019
Objective was to study genetic markers of thrombophilia in patients with hemophilia, which can affect the course of the disease and contribute to thrombotic complications. Material and methods.
T. Yu. Polyanskaya, I. S. Fevraleva, E. E. Karpov, N. V. Sadykova, M. S. Sampiev, A. V. Golobokov, G. V. Mishin, D. Yu. Petrovskiy, A. A. Koroleva, G. M. Galstyan, A. B. Sudarikov, V. Yu. Zorenko   +11 more
doaj   +1 more source

Discrepant ratios of arterial versus venous thrombosis in hemophilia A as compared with hemophilia B

, 2014
The occurrence of thrombosis in patients with congenital bleeding disorders represents an exceptional event. Hemophilia A and hemophilia B patients have been showed to present both arterial and venous thrombosis (85 cases of arterial thrombosis and 34 ...
Irene Bertozzi, Bertozzi, Irene, De Marinis, Giulia Berti, Sambado, Luisa, Antonio Girolami, Girolami, Antonio, Luisa Sambado, Giulia Berti de Marinis, Tasinato, Valentina, Valentina Tasinato   +9 more
core   +1 more source

Prime editing in neuropsychiatric disorders: From mutation‐specific target selection to clinical translation

Neuroprotection, EarlyView.
Abstract Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)‐based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR‐associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)‐encoded template.
Tianshan Ji, Yuan Zhang, Jinyi Zhao, Yiping Lu, Chengkun Wang   +4 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

Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques [PDF]

, 2002
The feasibility, safety, and efficacy of liver-directed gene transfer was evaluated in 5 male macaques (aged 2.5 to 6.5 years) by using a recombinant adeno-associated viral (rAAV) vector (rAAV-2 CAGG-hFIX) that had previously mediated persistent ...
Hanawa, H, Ng, CYC, Mandrell, TD, Vanin, EF, Nathwani, AC, Hu, Y, Davidoff, AM, Hoffer, FA, Zhou, J, Slaughter, C, Nikanorov, A, Lozier, JN, Nienhuis, AW   +12 more
core  

Gene Therapy Approaches for the Treatment of Hemophilia B

, 2023
In contrast to the standard enzyme-replacement therapy, administered from once per 7–14 days to 2–3 times a week in patients with severe hemophilia B, as a result of a single injection, gene therapy can restore F9 gene expression and maintain
Sofya G. Feoktistova, Pavel Y. Volchkov, Anastasiia B. Soroka, Olga N. Mityaeva   +3 more
core   +1 more source

A Rare Case of Acquired Haemophilia A Following Sigmoid Adenocarcinoma: Case Report and Literature Review

Asia-Pacific Journal of Clinical Oncology, EarlyView.
Acquired haemophilia A (AHA) is a rare complication of malignancy. We report a case of AHA secondary to colorectal cancer which presented as post‐operative bleeding, progressive activated partial thromboplastin time prolongation, precipitous factor VIII (FVIII) decline and inhibitor development.
Benjamin Reardon, Harini Haran, Jennifer Curnow   +2 more
wiley   +1 more source

The Molecular Basis of FIX Deficiency in Hemophilia B

, 2022
Coagulation factor IX (FIX) is a vitamin K dependent protein and its deficiency causes hemophilia B, an X-linked recessive bleeding disorder. More than 1000 mutations in the F9 gene have been identified in hemophilia B patients.
Weikai Li, Guomin Shen, Meng Gao, Qing Cao   +3 more
core   +1 more source