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Transplanting FVIII/ET3-secreting cells in fetal sheep increases FVIII levels long-term without inducing immunity or toxicity.
Rodriguez M, Trevisan B, Ramamurthy RM, George SK, Diaz J, Alexander J, Meares D, Schwahn DJ, Quilici DR, Figueroa J, Gautreaux M, Farland A, Atala A, Doering CB, Spencer HT, Porada CD, Almeida-Porada G. Rodriguez M, et al. Among authors: porada cd. Nat Commun. 2023 Jul 14;14(1):4206. doi: 10.1038/s41467-023-39986-1. Nat Commun. 2023. PMID: 37452013 Free PMC article.
Thus, these studies attest to the feasibility, immunologic advantage, and safety of treating hemophilia A prior to birth....
Thus, these studies attest to the feasibility, immunologic advantage, and safety of treating hemophilia A prior to birth....
Autologous bone marrow-derived MSCs engineered to express oFVIII-FLAG engraft in adult sheep and produce an effective increase in plasma FVIII levels.
Trevisan B, Rodriguez M, Medder H, Lankford S, Combs R, Owen J, Atala A, Porada CD, Almeida-Porada G. Trevisan B, et al. Among authors: porada cd. Front Immunol. 2022 Dec 2;13:1070476. doi: 10.3389/fimmu.2022.1070476. eCollection 2022. Front Immunol. 2022. PMID: 36532079 Free PMC article.
METHODS: We tested, in a highly translational large animal (sheep) model, whether the unique immunological and biological properties of autologous bone marrow (BM)-derived mesenchymal stromal cells (MSCs) could enable them to serve as cellular delivery vehicles to provide long- …
METHODS: We tested, in a highly translational large animal (sheep) model, whether the unique immunological and biological properties of auto …
Ionizing Particle Radiation as a Modulator of Endogenous Bone Marrow Cell Reprogramming: Implications for Hematological Cancers.
Muralidharan S, Sasi SP, Zuriaga MA, Hirschi KK, Porada CD, Coleman MA, Walsh KX, Yan X, Goukassian DA. Muralidharan S, et al. Among authors: porada cd. Front Oncol. 2015 Oct 14;5:231. doi: 10.3389/fonc.2015.00231. eCollection 2015. Front Oncol. 2015. PMID: 26528440 Free PMC article. Review.
Inefficient DNA repair processes in HSC and early hematopoietic progenitors can lead to an accumulation of mutations whereas long-lasting oxidative stress can impair hematopoiesis itself, thereby causing long-term damage to hematopoietic cells in the BM niche. We report he …
Inefficient DNA repair processes in HSC and early hematopoietic progenitors can lead to an accumulation of mutations whereas long-lasting ox …
Hemophilia A: an ideal disease to correct in utero.
Porada CD, Rodman C, Ignacio G, Atala A, Almeida-Porada G. Porada CD, et al. Front Pharmacol. 2014 Dec 11;5:276. doi: 10.3389/fphar.2014.00276. eCollection 2014. Front Pharmacol. 2014. PMID: 25566073 Free PMC article. Review.
There is thus a need for novel treatments, such as those using stem cells and/or gene therapy, which have the potential to mediate long-term correction or permanent cure following a single intervention. ...
There is thus a need for novel treatments, such as those using stem cells and/or gene therapy, which have the potential to mediate long-t …
Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene Delivery.
Porada CD, Almeida-Porada G. Porada CD, et al. J Genet Syndr Gene Ther. 2012 May 25;S1:011. doi: 10.4172/2157-7412.S1-011. J Genet Syndr Gene Ther. 2012. PMID: 23264887 Free PMC article.
There is thus a need for novel treatments that can promise long-term or permanent correction. In contrast to existing protein based therapeutics, gene therapy offers to provide a permanent cure following few, or even a single, treatment. ...
There is thus a need for novel treatments that can promise long-term or permanent correction. In contrast to existing protein based t …
In vivo generation of beta-cell-like cells from CD34(+) cells differentiated from human embryonic stem cells.
Goodrich AD, Ersek A, Varain NM, Groza D, Cenariu M, Thain DS, Almeida-Porada G, Porada CD, Zanjani ED. Goodrich AD, et al. Among authors: porada cd. Exp Hematol. 2010 Jun;38(6):516-525.e4. doi: 10.1016/j.exphem.2010.03.002. Epub 2010 Mar 12. Exp Hematol. 2010. PMID: 20227460 Free PMC article.
An average of 1.51 ng/mL human C-peptide was detected in serum from eight animals transplanted with differentiated cell populations and assayed up to 55 months posttransplantation. Transplantation of as few as 23,500 cells resulted in long-term sustainable beta-cell-like a …
An average of 1.51 ng/mL human C-peptide was detected in serum from eight animals transplanted with differentiated cell populations and assa …
The sheep model of in utero gene therapy.
Porada CD, Park P, Almeida-Porada G, Zanjani ED. Porada CD, et al. Fetal Diagn Ther. 2004 Jan-Feb;19(1):23-30. doi: 10.1159/000074255. Fetal Diagn Ther. 2004. PMID: 14646413 Review.
Performing GT in utero would allow early correction prior to disease onset and is thus one of the few therapeutic modalities that could promise the birth of a healthy infant. Several features of the developing fetus may circumvent obstacles that have thus far been observed …
Performing GT in utero would allow early correction prior to disease onset and is thus one of the few therapeutic modalities that could prom …
Transduction of long-term-engrafting human hematopoietic stem cells by retroviral vectors.
Porada CD, Tran ND, Almeida-Porada G, Glimp HA, Pixley JS, Zhao Y, Anderson WF, Zanjani ED. Porada CD, et al. Hum Gene Ther. 2002 May 1;13(7):867-79. doi: 10.1089/10430340252899037. Hum Gene Ther. 2002. PMID: 11975852
In the present studies, we used the fetal sheep xenograft model of human hematopoiesis to evaluate whether human long-term engrafting HSCs could be transduced in vivo, within a fetal microenvironment. ...Transgene expression persisted throughout the serial transplantation. …
In the present studies, we used the fetal sheep xenograft model of human hematopoiesis to evaluate whether human long-term engrafting …
Induction of stable prenatal tolerance to beta-galactosidase by in utero gene transfer into preimmune sheep fetuses.
Tran ND, Porada CD, Almeida-Porada G, Glimp HA, Anderson WF, Zanjani ED. Tran ND, et al. Among authors: porada cd. Blood. 2001 Jun 1;97(11):3417-23. doi: 10.1182/blood.v97.11.3417. Blood. 2001. PMID: 11369632 Free article.
The successful transduction of hematopoietic stem cells and long-term (28 months) transgene expression within the hematopoietic system following the direct injection of high-titer retroviral vectors into preimmune fetal sheep was previously demonstrated. ...
The successful transduction of hematopoietic stem cells and long-term (28 months) transgene expression within the hematopoietic syste …
Cotransplantation of human stromal cell progenitors into preimmune fetal sheep results in early appearance of human donor cells in circulation and boosts cell levels in bone marrow at later time points after transplantation.
Almeida-Porada G, Porada CD, Tran N, Zanjani ED. Almeida-Porada G, et al. Among authors: porada cd. Blood. 2000 Jun 1;95(11):3620-7. Blood. 2000. PMID: 10828053 Free article.
The results presented here demonstrate that the cotransplantation of both autologous and allogeneic human bone marrow-derived stromal cell progenitors resulted in an enhancement of long-term engraftment of human cells in the bone marrow of the chimeric animals and in earli …
The results presented here demonstrate that the cotransplantation of both autologous and allogeneic human bone marrow-derived stromal cell p …
13 results