Results 291 to 300 of about 2,414,789 (346)

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

Advanced Functional Materials, EarlyView.
Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

Hyperglycemic Hyperosmolar State as the Initial Presentation of Wolfram Syndrome: A Common Complication Revealing a Rare Disease-A Case Report. [PDF]

Clin Case Rep
Ingawale S, Shinde I, Rachakonda ST, Pandey A, Shah A.   +4 more
europepmc   +1 more source

Smart, Bio‐Inspired Polymers and Bio‐Based Molecules Modified by Zwitterionic Motifs to Design Next‐Generation Materials for Medical Applications

Advanced Functional Materials, EarlyView.
Bio‐based and (semi‐)synthetic zwitterion‐modified novel materials and fully synthetic next‐generation alternatives show the importance of material design for different biomedical applications. The zwitterionic character affects the physiochemical behavior of the material and deepens the understanding of chemical interaction mechanisms within the ...
Theresa M. Lutz, Jonas De Breuck, Sahar Salehi, Meike N. Leiske   +3 more
wiley   +1 more source

Advancing rare disease therapeutics through digital twins: Opportunities in drug development and precision dosing. [PDF]

Comput Struct Biotechnol J
Dette CMU, Alberg V, Rüdesheim S, Selzer D, Marok FZ, Bragazzi NL, Fuhr LM, Brunak S, Pearson ER, Zahn T, Kiritsi D, Schwab M, Lehr T.   +12 more
europepmc   +1 more source

Correction: Network Meta-analysis of Ravulizumab and Alternative Interventions for the Treatment of Neuromyelitis Optica Spectrum Disorder

Neurology and Therapy
Stacey L. Clardy, Sean J. Pittock, Orhan Aktas, Jin Nakahara, Noriko Isobe, Diego Centonze, Sami Fam, Adrian Kielhorn, Jeffrey C. Yu, Jeroen Jansen, Ina Zhang   +10 more
doaj   +1 more source

An optimized variant prioritization process for rare disease diagnostics: recommendations for Exomiser and Genomiser. [PDF]

Genome Med
Cooperstein IB, Marwaha S, Ward A, Kobren SN, Carter JN, Undiagnosed Diseases Network, Wheeler MT, Marth GT.   +7 more
europepmc   +1 more source

Multi-criteria decision analysis of value evaluation of rare disease medical insurance drugs in China. [PDF]

BMC Public Health
Chen K, Duan Y, Cao Z, Wu Y, Wang J, Jiang B, Gu R.   +6 more
europepmc   +1 more source

Optimizing recruitment in rare disease research: a cross-sectional online study evaluating sampling strategies for hard-to-reach populations. [PDF]

Orphanet J Rare Dis
Baffour S, Löwe B, Braun A, Uhlenbusch N.   +3 more
europepmc   +1 more source

Community Pharmacists’ Awareness and Perceptions on the Role of Pharmacists in the Treatment and Management of Rare Disease Patients in Korea

Jeongeun Kim, Sung Cil Lim
openalex   +2 more sources