Results 91 to 100 of about 105,245 (307)

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

SURMOUNT‐REAL UK: A Pragmatic Randomized Clinical Trial to Assess the Effectiveness of Tirzepatide in Adults With Obesity

Obesity, EarlyView.
ABSTRACT Objective SURMOUNT‐REAL UK will evaluate the effectiveness of tirzepatide when offered in addition to standard‐of‐care (SoC) in adults with Class I obesity (BMI ≥ 30 and ≤ 34.9 kg/m2) and without diabetes in a UK primary care setting. Methods A 5‐year, phase 4, multicenter, open‐label, pragmatic randomized clinical trial is enabled through ...
Martin K. Rutter, Julie Mount, J. Martin Gibson, Joanne Webb, Cheryl Marler, Catherine Reed, Mugdha Gore, Wenyu Ye, Elisa Gomez Valderas, John Wilding, Naveed Sattar, Melanie J. Davies, Kamlesh Khunti, Richard Haynes, Rachel L. Batterham   +14 more
wiley   +1 more source

Hydroxyurea mobile directly observed therapy versus standard monitoring in patients with sickle cell anemia: a phase 2 randomized trial

Communications Medicine
Background Sickle cell anemia (SCA) prevalence remains high in sub-Saharan Africa. Long-term treatment with hydroxyurea (HU) increases survival, however, poor adherence to treatment could limit effectiveness.
Philip Sasi, Abel Makubi, Raphael Z. Sangeda, Mariam Y. Ngaeje, Bruno P. Mmbando, Joseph Soka, Caterina Rosano, Alex S. Magesa, Sharon E. Cox, Julie Makani, Enrico M. Novelli   +10 more
doaj   +1 more source

The Reliability of Intraoperative Frozen Section for Glottic Lesions Suspected of Early Malignancy

Otolaryngology–Head and Neck Surgery, EarlyView.
Abstract Objective Glottic lesions suspected of malignancy may be treated by laser cordectomy. Although obviating the preoperative biopsy can expedite treatment and preserve the surgical planes, it poses a risk of mistreatment. This study sought to determine if intraoperative frozen section (FS) prior to cordectomy can reliably guide lesion management.
Gal Levi, Yaniv Hamzany, Nir Tsur, Yulia Strenov, Daniel Ben‐Ner, Hagit Shoffel‐Havakuk   +5 more
wiley   +1 more source

Variations in mitochondrial genome as potential prognostic markers in sickle cell disease

Haematologica
Alterations in the mitochondrial genome integrity, including changes in mitochondrial DNA copy number (mtDNA-CN) and accumulation of mtDNA mutations, are associated with aging and diverse disorders, often linked to underlying systemic inflammation and ...
Rudra Ray, Haiou Li, Shijinqiu Gao, Nancy Asomaning, Kang Le, Maliha M. Ahmad, Xunde Wang, Yuesheng Li, Sayuri Kamimura, Clifton L. Dalgard, Chengyu Liu, Zenaide M.N. Quezado, Justin Lack, Laxminath Tumburu, Swee Lay Thein   +14 more
doaj   +1 more source

Prenatal Exome Sequencing Identifies Dual Maternal‐Fetal Diagnosis of HbF Mission Bay, a Novel HBG2 Variant Associated With Methemoglobinemia, Hypoxia and Hemolytic Anemia

Prenatal Diagnosis, EarlyView.
ABSTRACT Prenatal exome sequencing (ES) can establish rare genetic diagnoses in a fetus but may also lead to occult genetic diagnosis in a biological parent. We present a case of dual fetal and maternal diagnosis by prenatal ES, in a fetus with unexplained anemia and in a pregnant patient with sickle cell disease (SCD) and recurrent unexplained hypoxia.
Matthew A. Shear, Billie Lianoglou, Ugur Hodoglugil, W. Patrick Devine, Ashutosh Lal, Juan Gonzalez, Teresa N. Sparks   +6 more
wiley   +1 more source

Attitudes Toward Prenatal Interventions in the Fanconi Anemia Community

Prenatal Diagnosis, EarlyView.
ABSTRACT Objective In‐utero cell and gene therapies may offer prenatal treatment options for inherited diseases. Preclinical data suggests in‐utero (IU) hematopoietic stem cell transplantation (HSCT) could prevent Fanconi anemia (FA) related bone marrow failure without genotoxic conditioning or immune suppression.
Tony Lum, Catherine Lee, Tippi MacKenzie, Billie Lianoglou, Agnieszka Czechowicz   +4 more
wiley   +1 more source

The Bloodline newsletter : Sickle Cell Data Collection (SCDC) Program quarterly newsletter ; Summer 2019

Mission: To improve quality of life, life expectancy, and health among people living with sickle cell disease (SCD)

core   +1 more source

In Utero HSC Transplantation for Sickle Cell Disease: A Potential Therapeutic Approach That Overcomes Complications of Current Therapies

Prenatal Diagnosis, EarlyView.
ABSTRACT Sickle cell disease (SCD) affects millions worldwide but has limited treatment options, most of which carry significant side effects. At present, the only curative treatment for SCD is allogeneic or gene‐modified autologous hematopoietic stem cell (HSC) transplantation (Tx).
Oluwaseun O. Babatunde, Madeleine G. Bibby, Anthony Atala, Graça Almeida‐Porada, Christopher D. Porada   +4 more
wiley   +1 more source

P-021: THE LIVING WELL WITH SICKLE CELL MOBILE APPLICATION

HemaSphere, 2022
MATTHEWS A., MATTHEWS M.
doaj   +1 more source