Adeno-associated virus (AAV) vectors are promising candidates for gene therapy. However, a number of recent preclinical large animal studies failed to translate into the clinic. This illustrates the formidable challenge of choosing the animal models that
Estrella Lopez-Gordo +6 more
doaj +1 more source
Multiple gene editing in porcine embryos using a combination of microinjection, electroporation, and transfection methods [PDF]
Background and Aim: Mosaicism – the presence of both wild-type and mutant alleles – is a serious problem for zygotic gene modification through gene editing using the Clustered regularly interspaced short palindromic repeats-Cas9 (CRISPR/ Cas9) system ...
Quynh Anh Le +8 more
doaj +1 more source
The feline AB blood group system (blood types A, B, and AB) encoding the cytidine monophosphate-N-acetylneuraminic acid hydroxylase (CMAH) gene is the most significant in transfusion medicine and hemolysis of the newborn for cats.
Yumiko Uno +10 more
doaj +1 more source
The sugar molecule N-glycolylneuraminic acid (Neu5Gc) is one of the most common sialic acids discovered in mammals. Cytidine monophospho-N-acetylneuraminic acid hydroxylase (CMAH) catalyses the conversion of N-acetylneuraminic acid (Neu5Ac) to Neu5Gc ...
Oluwamayowa Joshua Ogun +3 more
doaj +1 more source
Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy. [PDF]
Cardiac failure is a major cause of mortality in patients with Duchenne muscular dystrophy (DMD). Antisense-mediated exon skipping has the ability to correct out-of-frame mutations in DMD to produce truncated but functional dystrophin.
Alison M Blain +11 more
doaj +1 more source
Donor-Derived Cell-Free DNA (dd-cfDNA) as an Early Noninvasive Biomarker of Graft Injury in Pig-to-Monkey Islet Xenotransplantation. [PDF]
ABSTRACT Background In pig‐to‐nonhuman primate islet transplantation, reliable, sensitive biomarkers are needed to detect graft damage at an early stage before irreversible islet loss occurs. In our study, we investigated donor‐derived cell‐free DNA (dd‐cfDNA) as an early, noninvasive biomarker of graft injury by analyzing its correlation with porcine ...
Yan JJ +10 more
europepmc +2 more sources
Early Experimental Report of the First 8-Gene-Edited Pig-to-Rhesus Macaque Cardiac Xenotransplantation in China. [PDF]
ABSTRACT Objectives This study aimed to investigate the functional recovery of an 8‐gene‐edited pig heart after orthotopic xenotransplantation, the characteristics of the recipient's immune responses, and the efficacy of postoperative complication management to provide comprehensive experimental evidence for the clinical translation of xenogeneic ...
Wang X +17 more
europepmc +2 more sources
Porcine kidney xenotransplantation: From primate models to clinical reality. [PDF]
In the face of a critical shortage of human donor kidneys for end‐stage renal disease patients, porcine kidney xenotransplantation has emerged as a viable solution. This field has navigated major hurdles, including immune rejection, physiological incompatibilities, potential biomechanical differences and the risk of cross‐species infection. To overcome
Guo Z, Zhang L, Deng S, Qin C.
europepmc +2 more sources
An Overview of the Importance and Value of Porcine Species in Sialic Acid Research
Humans frequently interact with pigs, whose meat is also one of the primary sources of animal protein. They are one of the main species at the center of sialic acid (Sia) research.
Oluwamayowa Joshua Ogun +2 more
doaj +1 more source
Improved ESI-MS Sensitivity via an Imidazolium Tag (DAPMI-ITag) for Precise Sialic Acid Detection in Human Serum and CMAH-Null Mouse Tissues. [PDF]
Sialic acids (Sias), consisting primarily of N-acetylneuraminic acid (Neu5Ac) and N-glycolylneuraminic acid (Neu5Gc), play crucial roles in many biological processes. The detection and quantification of Sias are essential for understanding their roles in
Zhang YY +6 more
europepmc +2 more sources

