Results 161 to 170 of about 204,284 (265)
CRISPR‐Cas9 and precision editing technologies enable a variant‐mechanism‐driven framework for genetic disease research and therapeutic development. Pathogenic variants are first interpreted according to mutation type, coding or regulatory consequence, tissue context, and disease mechanism.
Zijing Wen, Jianming Su
wiley +1 more source
ABSTRACT Background and Aim Children with sickle cell disease (SCD) in low‐ and middle‐income countries (LMICs) face significant disease‐related challenges and socioeconomic status (SES) disparities that may negatively impact their academic performance. This systematic review synthesized existing evidence on academic performance among children with SCD
Shubaya K. Naggayi +7 more
wiley +1 more source
Sickle cell trait and diabetes tests : what every healthcare provider should know [PDF]
According to the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), the hemoglobin A1C assay may be unreliable for diagnosing and monitoring diabetes and prediabetes in individuals with sickle cell trait (SCT) or other ...
core
Strategies and mechanisms of precision genome engineering: From gene editing to genome writing
In this review, we examined the progression of genome manipulation from stochastic nuclease‐mediated cutting toward precise editing and programmable genome writing. We discussed tools like multi‐kilobase RNA‐guided integrators and Artificial Intelligence (AI)‐designed effectors and showed how these advances enable researchers to treat genomes as ...
Kerui Huang +19 more
wiley +1 more source
The Patient with Sickle Hemoglobin
Robert L. Richardson, Emily Taylor
openaire +1 more source
American Journal of Hematology, Volume 101, Issue 9, Page 2430-2434, September 2026.
Ferras Alashkar +10 more
wiley +1 more source
Abstract Accurate drug concentration measurement is essential for precision pharmacotherapy, but conventional therapeutic drug monitoring (TDM) requires venous sampling, increasing patient burden, and potentially limiting participation in TDM and model‐informed precision dosing (MIPD).
Hari Prabhath Tummala +5 more
wiley +1 more source
Genome Editing for Glycogen Storage Diseases
ABSTRACT Gene therapy has been developed for several glycogen storage diseases and has advanced into clinical trials. However, the limitations of these gene therapies with regard to stability following treatment early in life have led to the development of genome editing.
Troy von Beck +2 more
wiley +1 more source
Cerebral Blood Transit in Sickle Cell Anemia
ABSTRACT Background Sickle cell anemia (SCA) patients upregulate cerebral blood flow to compensate for decreased arterial oxygen content. Such hyperemic conditions can manifest as venous hyperintense signal on arterial spin labeling (ASL) MRI, which may reflect faster capillary blood transit, altered oxygen extraction fraction (OEF), and infarct risk ...
Wesley T. Richerson +10 more
wiley +1 more source
Smart Design: Integrating Artificial Intelligence and Gene Editing for Advanced mRNA Therapeutics
The challenges of mRNA therapy and the application of artificial intelligence and gene editing in the field of mRNA drugs. ABSTRACT Artificial intelligence (AI) and gene editing are increasingly being applied to the design and evaluation of mRNA therapeutics.
Haixing Shi +11 more
wiley +1 more source

