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Current progress in CRISPR-Cas systems for rare diseases.
Prog Mol Biol Transl Sci
January 2025
Department of Biotechnology, Faculty of Engineering and Technology, Rama University, Kanpur, Uttar Pradesh, India. Electronic address:
The groundbreaking CRISPR-Cas gene editing method permits exact genetic code alteration. The "CRISPR" DNA protects bacteria from viruses. CRISPR-Cas utilizes a guide RNA to steer the Cas enzyme to the genome's gene editing target.
View Article and Find Full Text PDFAdult-type ALCAPA: missed at preoperative evaluation.
Indian J Thorac Cardiovasc Surg
February 2025
Ankara City Hospital Cardiovascular Surgery, Ankara, Turkey.
Unlabelled: The Bland-White-Garland syndrome, or Anomalous Origin of the Left Coronary Artery from the Pulmonary Artery (ALCAPA) syndrome, is a rare congenital cardiac anomaly often associated with high mortality, if left untreated. We present a case of a 43-year-old female with undiagnosed ALCAPA who initially underwent mitral valve surgery for severe mitral regurgitation, only to require reoperation due to adult-type ALCAPA. Intraoperatively, the discovery of dilated right coronary artery and its branches and absence of the left coronary ostium prompted further investigation, leading to the diagnosis of adult-type ALCAPA.
View Article and Find Full Text PDFThe effect of different root canal irrigations on the accuracy of apex locators: A systematic review.
J Clin Exp Dent
December 2024
Endodontics, Tehran, Iran.
Background: For a successful root canal treatment, it is necessary to determine the correct working length. One of the most used tools to determine the length is: radiography and apex locator. The accuracy of APEX locator of different generations depends on various factors.
View Article and Find Full Text PDFNeurovisual Training With Acoustic Feedback: An Innovative Approach for Nystagmus Rehabilitation.
Arch Rehabil Res Clin Transl
December 2024
Section of Neurorehabilitation, Department of Translational Research and New Technologies in Medicine and Surgery, University of Pisa, Pisa, Italy.
Nystagmus has various clinical manifestations, including downbeat, upbeat, and torsional types, each associated with distinct neurologic features. Current rehabilitative interventions focusing on fixation training and optical correction often fail to achieve complete resolution. When nystagmus coexists with fragile X-associated tremor/ataxia syndrome (FXTAS), functional impairments worsen, particularly affecting balance.
View Article and Find Full Text PDFCost-consequence analysis of early vs. delayed natalizumab use in highly active relapsing-remitting multiple sclerosis: a simulation study.
J Neurol
January 2025
Department of Neurology, Center of Clinical Neuroscience, University Hospital Carl Gustav Carus, Technische Universität Dresden, Fetscherstr. 74, 01307, Dresden, Germany.
Background: Natalizumab (NAT) is an established disease-modifying therapy (DMT) for highly active multiple sclerosis (MS). However, its use involves complex decision-making, often leading to initial use of lower efficacy therapies. Recently, the first biosimilar NAT was approved, enabling competitive pricing.
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