Therapeutic mesenchymal stromal cells for immunotherapy and for gene and drug delivery

G Almeida-Porada, AJ Atala, CD Porada - Molecular Therapy-Methods & …, 2020 - cell.com
Mesenchymal stromal cells (MSCs) possess several fairly unique properties that, when
combined, make them ideally suited for cellular-based immunotherapy and as vehicles for …

Past, present and future of ICSI in livestock species

O Briski, DF Salamone - Animal Reproduction Science, 2022 - Elsevier
During the past 2 decades, intracytoplasmic sperm injection (ICSI) has become a routine
technique for clinical applications in humans. The widespread use among domestic species …

Anti-factor IXa/X bispecific antibody ACE910 prevents joint bleeds in a long-term primate model of acquired hemophilia A

A Muto, K Yoshihashi, M Takeda… - Blood, The Journal …, 2014 - ashpublications.org
ACE910 is a humanized anti-factor IXa/X bispecific antibody mimicking the function of factor
VIII (FVIII). We previously demonstrated in nonhuman primates that a single IV dose of …

Intracytoplasmic sperm injection in domestic and wild mammals

DF Salamone, NG Canel, MB Rodríguez - Reproduction, 2017 - rep.bioscientifica.com
Intracytoplasmic sperm injection (ICSI) has become a useful technique for clinical
applications in the horse-breeding industry. However, both ICSI blastocyst and offspring …

Protein-engineered coagulation factors for hemophilia gene therapy

BJ Samelson-Jones, VR Arruda - Molecular Therapy-Methods & Clinical …, 2019 - cell.com
Hemophilia A (HA) and hemophilia B (HB) are X-linked bleeding disorders due to
inheritable deficiencies in either coagulation factor VIII (FVIII) or factor IX (FIX), respectively …

In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application

G Almeida-Porada, A Atala, CD Porada - Molecular Therapy-Methods & …, 2016 - cell.com
Recent advances in high-throughput molecular testing have made it possible to diagnose
most genetic disorders relatively early in gestation with minimal risk to the fetus. These …

Animal models of hemophilia

DE Sabatino, TC Nichols, E Merricks… - Progress in molecular …, 2012 - Elsevier
The X-linked bleeding disorder hemophilia is caused by mutations in coagulation factor VIII
(hemophilia A) or factor IX (hemophilia B). Unless prophylactic treatment is provided …

Large animal models of rare genetic disorders: sheep as phenotypically relevant models of human genetic disease

AR Pinnapureddy, C Stayner, J McEwan… - Orphanet journal of rare …, 2015 - Springer
Animals that accurately model human disease are invaluable in medical research, allowing
a critical understanding of disease mechanisms, and the opportunity to evaluate the effect of …

Gene modification therapies for hereditary diseases in the fetus

CNZ Mattar, JKY Chan, M Choolani - Prenatal Diagnosis, 2023 - Wiley Online Library
Proof‐of‐principle disease models have demonstrated the feasibility of an intrauterine gene
modification therapy (in utero gene therapy (IUGT)) approach to hereditary diseases as …

Transplanting FVIII/ET3-secreting cells in fetal sheep increases FVIII levels long-term without inducing immunity or toxicity

M Rodriguez, B Trevisan, RM Ramamurthy… - Nature …, 2023 - nature.com
Hemophilia A is the most common X-linked bleeding disorder affecting more than half-a-
million individuals worldwide. Persons with severe hemophilia A have coagulation FVIII …