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Journal Articles

Enhancing donor cell engraftment in prenatal hematopoietic stem cell transplantation with novel antibody-drug conjugates

Oluwaseun Babatunde, Madeleine Bibby, Walaa Mohamed, Martin Rodriguez, Nicholas Edenhoffer, Jonathan Diaz, Bill Vaughan, Anthony Atala, Christopher Doering, H. Trent Spencer, Christopher Porada, M. Graca Almeida-Porada

Blood (2025) 146 (Supplement 1): 4086.

https://doi.org/10.1182/blood-2025-4086

Published: 2025

Journal Articles

Transduction efficiency and vector biodistribution following prenatal vs neonatal AAV5 gene therapy for Hemophilia A in a sheep model

Martin Rodriguez, Walaa Mohamed, Oluwaseun Babatunde, Bill Vaughan, Andrew Farland, John Owen, Christopher Doering, H. Trent Spencer, Anthony Atala, Christopher Porada, M. Graca Almeida-Porada

Blood (2025) 146 (Supplement 1): 4316.

https://doi.org/10.1182/blood-2025-4316

Published: 2025

Journal Articles

Prenatal transplantation of a clinical cell product engineered to secrete FVIII in a sheep with Hemophilia A corrects the severe bleeding phenotype and prevents hemarthroses

M. Graca Almeida-Porada, Walaa Mohamed, Martin Rodriguez, Oluwaseun Babatunde, Ritu Ramamurthy, Qi Xing, Vladimir Mashanov, Bill Vaughan, Jordan Shields, Andrew Farland, Christopher Doering, H. Trent Spencer, Anthony Atala, Christopher Porada

Blood (2025) 146 (Supplement 1): 1064.

https://doi.org/10.1182/blood-2025-1064

Published: 2025

Journal Articles

AAV delivers as a lingering guest, not an invader

Graça Almeida-Porada

Blood (2024) 143 (23): 2341–2342.

https://doi.org/10.1182/blood.2024024506

Published: 2024

Journal Articles

Novel Sheep Model of Sickle Cell Disease Reproduces Human Clinical and Laboratory Parameters

Caroline E. Kuczynski, Iuri V. Perisse, Misha Regouski, Elmira Alipour, Cecily LaPradd, Ying Liu, Michael W. Beaty, Daniel B. Kim-Shapiro, Anthony Atala, Irina Polejaeva, Christopher D. Porada, M. Graca Almeida-Porada

Blood (2022) 140 (Supplement 1): 8220.

https://doi.org/10.1182/blood-2022-168050

Published: 2022

Journal Articles

Using a Human Liver Tissue Equivalent (hLTE) Platform to Define the Functional Impact of Liver-Directed AAV Gene Therapy

Ritu M Ramamurthy, Wen Ting Zheng, Sunil George, Meimei Wan, Yu Zhou, Baisong Lu, Colin E Bishop, Anthony Atala, Christopher D Porada, M. Graca Almeida-Porada

Blood (2021) 138 (Supplement 1): 2938.

https://doi.org/10.1182/blood-2021-147917

Published: 2021

Journal Articles

In Utero Transplantation of FVIII-Expressing Human Placental Cells Upregulates Gene Pathways Associated with Immune Tolerance, Altering the Response to Postnatal FVIII Infusion

Martin Rodriguez, Brady Trevisan, Sunil George, Jordan E Shields, Jorge Figueroa, Diane Meares, John Owen, Anthony Atala, Christopher B Doering, H. Trent Spencer, Christopher D Porada, M. Graca Almeida-Porada

Blood (2021) 138 (Supplement 1): 1863.

https://doi.org/10.1182/blood-2021-153417

Published: 2021

Journal Articles

Administration of FVIII-Expressing Human Placental Cells to Juvenile Sheep Yields Multi-Organ Engraftment, Therapeutic Plasma FVIII Levels and Alter Immune Signaling Pathways to Evade FVIII Inhibitor Induction

Brady Trevisan, Martin Rodriguez, Jacqueline Dizon, Sunil George, Jordan E Shields, Shannon Lankford, Rebecca Combs, John Owen, Anthony Atala, Christopher B Doering, H. Trent Spencer, Christopher D Porada, M. Graca Almeida-Porada

Blood (2021) 138 (Supplement 1): 3966.

https://doi.org/10.1182/blood-2021-151207

Published: 2021

Journal Articles

A new “FIX” for hemophilia B gene therapy

Graça Almeida-Porada

Blood (2021) 137 (21): 2860–2861.

https://doi.org/10.1182/blood.2021011753

Published: 2021

Journal Articles

Evaluation of Maternal Safety Following Prenatal Cell and Gene Therapy for Hemophilia a in a Large Animal Model Demonstrates Absence of Maternal Exposure to the Cells or Gene Products Infused into the Fetus

Quan DM Pham, Martin Rodriguez, BSc, Ritu M Ramamurthy, Sunil George, PhD, Jorge Figueroa, MD, Anthony Atala, MD, Christopher B Doering, PhD, H. Trent Spencer, PhD, Christopher D Porada, PhD, M. Graca Almeida-Porada, MD PhD

Blood (2020) 136 (Supplement 1): 32.

https://doi.org/10.1182/blood-2020-140883

Published: 2020

Journal Articles

Administration of Cells Engineered to Secrete Fviii-mcoET3 in Prenatal Sheep Recipients Results in Sustained Curative Fviii Plasma Levels for 3 Years after Birth, without Immune or Toxicity-Related Adverse Events

Martin Rodriguez, BSc, Brady Trevisan, Sunil George, PhD, Ritu M Ramamurthy, Andrew Rabah, Jordan E Shields, MS, Denise Schwahn, DVM, Jorge Figueroa, MD, Diane Meares, John Owen, MDMBA,FRCPC, Michael Gautreaux, PhD, D.ABHI, Anthony Atala, MD, Christopher B Doering, PhD, H. Trent Spencer, PhD, Christopher D Porada, PhD, M. Graca Almeida-Porada, MD PhD

Blood (2020) 136 (Supplement 1): 1–2.

https://doi.org/10.1182/blood-2020-140845

Published: 2020

Journal Articles

Delivery of Fviii-mcoET3 to Previously Untreated Sheep Using Human Placental Cells Enables Durable Elevation of Plasma FVIII Levels and Avoids Inhibitor Formation

Brady Trevisan, Martin Rodriguez, BSc, Sunil George, PhD, Shannon Lankford, Jordan E Shields, MS, Jorge Figueroa, MD, Diane Meares, John Owen, MDMBA,FRCPC, Anthony Atala, MD, Christopher B Doering, PhD, H. Trent Spencer, PhD, Christopher D Porada, PhD, M. Graca Almeida-Porada, MD PhD

Blood (2020) 136 (Supplement 1): 34.

https://doi.org/10.1182/blood-2020-140745

Published: 2020

Journal Articles

Recreating the Hematopoietic Microenvironment in Vitro Using Microfluidic “on-a-Chip” Technology Enables the Study of Interactions of Normal and Malignant HSPC with Specific Bone Marrow Niches

Sunil George, PhD, Julio Aleman, MS, M. Graca Almeida-Porada, MD PhD, Aleksander Skardal, PhD, Christopher D Porada, PhD

Blood (2017) 130 (Supplement 1): 3779.

https://doi.org/10.1182/blood.V130.Suppl_1.3779.3779

Published: 2017

Journal Articles

Prenatal Transplantation of Placental Cells Engineered to Express FVIII Leads to Corrective Plasma Levels of FVIII after Birth

M. Graca Almeida-Porada, MD PhD, Sunil George, PhD, Alshaima Morsi, MD, Nadia El-Akabawy, MD, Jorge Figueroa, Jordan E Shields, MS, Diane Meares, Andrew M. Farland, MD, Anthony Atala, MD, Christopher B Doering, PhD, H. Trent Spencer, PhD, Christopher D Porada, PhD

Blood (2017) 130 (Supplement 1): 361.

https://doi.org/10.1182/blood.V130.Suppl_1.361.361

Published: 2017

Journal Articles

A New Approach to Expand Cord Blood Derived Hematopoietic Stem Cells, Using Bioengineered Human Fetal Liver Tissue 3D-Constructs

Saloomeh Mokhtari, Pedro Baptista, Dipen Vyas, PhD, Charles Jordan Freeman, Emma Moran, PhD, Christopher D Porada, PhD, Shay Soker, PhD, Graca Almeida-Porada, MD PhD

Blood (2015) 126 (23): 3097.

https://doi.org/10.1182/blood.V126.23.3097.3097

Published: 2015

Journal Articles

A Human Bone Marrow-Derived Stromal Cell Population with Hemogenic Potential

Saloomeh Mokhtari, Evan Joseph Colletti, PhD, Chad Sanada, PhD, Zanetta S. Lamar, MD, Paul J Simmons, Anthony Atala, MD, Diane S Krause, MD PhD, Esmail D. Zanjani, PhD, Christopher D Porada, PhD, Graca Almeida-Porada, MD PhD

Blood (2015) 126 (23): 1201.

https://doi.org/10.1182/blood.V126.23.1201.1201

Published: 2015

Journal Articles

Defining Engraftment Patterns and Interactions Between Hematopoietic Stem Cells and Stromal Cells after in Utero Transplantation

Saloomeh Mokhtari, Christopher D Porada, PhD, Evan Joseph Colletti, PhD, Graca Almeida-Porada, MD PhD

Blood (2015) 126 (23): 1869.

https://doi.org/10.1182/blood.V126.23.1869.1869

Published: 2015

Journal Articles

RUNX1C-Mediated Reprogramming Of Human Bone Marrow Stromal Cells into Early Blood Progenitors

Weihong Yin, PhD, Christopher D Porada, PhD, Stephen Walker, PhD, Colin Bishop, PhD, Graca Almeida-Porada, MD, PhD

Blood (2013) 122 (21): 2463.

https://doi.org/10.1182/blood.V122.21.2463.2463

Published: 2013

Journal Articles

CD166+CD34+ Cells Exhibit Marked Functional Differences During Fetal and Adult Life

Saloomeh Mokhtari, Zanetta S. Lamar, MD, Chris Booth, Frank Marini, III, PhD, Christopher D Porada, PhD, Graca Almeida-Porada, MD, PhD

Blood (2013) 122 (21): 2435.

https://doi.org/10.1182/blood.V122.21.2435.2435

Published: 2013

Journal Articles

Optimization Of Vascular Niches To Increase Hematopoietic Engraftment

Saloomeh Mokhtari, Evan Colletti, PhD, Christopher D Porada, PhD, Graca Almeida-Porada, MD, PhD

Blood (2013) 122 (21): 4456.

https://doi.org/10.1182/blood.V122.21.4456.4456

Published: 2013

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Enhancing donor cell engraftment in prenatal hematopoietic stem cell transplantation with novel antibody-drug conjugates

Transduction efficiency and vector biodistribution following prenatal vs neonatal AAV5 gene therapy for Hemophilia A in a sheep model

Prenatal transplantation of a clinical cell product engineered to secrete FVIII in a sheep with Hemophilia A corrects the severe bleeding phenotype and prevents hemarthroses

AAV delivers as a lingering guest, not an invader

Novel Sheep Model of Sickle Cell Disease Reproduces Human Clinical and Laboratory Parameters

Using a Human Liver Tissue Equivalent (hLTE) Platform to Define the Functional Impact of Liver-Directed AAV Gene Therapy

In Utero Transplantation of FVIII-Expressing Human Placental Cells Upregulates Gene Pathways Associated with Immune Tolerance, Altering the Response to Postnatal FVIII Infusion

Administration of FVIII-Expressing Human Placental Cells to Juvenile Sheep Yields Multi-Organ Engraftment, Therapeutic Plasma FVIII Levels and Alter Immune Signaling Pathways to Evade FVIII Inhibitor Induction

A new “FIX” for hemophilia B gene therapy

Evaluation of Maternal Safety Following Prenatal Cell and Gene Therapy for Hemophilia a in a Large Animal Model Demonstrates Absence of Maternal Exposure to the Cells or Gene Products Infused into the Fetus

Administration of Cells Engineered to Secrete Fviii-mcoET3 in Prenatal Sheep Recipients Results in Sustained Curative Fviii Plasma Levels for 3 Years after Birth, without Immune or Toxicity-Related Adverse Events

Delivery of Fviii-mcoET3 to Previously Untreated Sheep Using Human Placental Cells Enables Durable Elevation of Plasma FVIII Levels and Avoids Inhibitor Formation

Recreating the Hematopoietic Microenvironment in Vitro Using Microfluidic “on-a-Chip” Technology Enables the Study of Interactions of Normal and Malignant HSPC with Specific Bone Marrow Niches

Prenatal Transplantation of Placental Cells Engineered to Express FVIII Leads to Corrective Plasma Levels of FVIII after Birth

A New Approach to Expand Cord Blood Derived Hematopoietic Stem Cells, Using Bioengineered Human Fetal Liver Tissue 3D-Constructs

A Human Bone Marrow-Derived Stromal Cell Population with Hemogenic Potential

Defining Engraftment Patterns and Interactions Between Hematopoietic Stem Cells and Stromal Cells after in Utero Transplantation

RUNX1C-Mediated Reprogramming Of Human Bone Marrow Stromal Cells into Early Blood Progenitors

CD166+CD34+ Cells Exhibit Marked Functional Differences During Fetal and Adult Life

Optimization Of Vascular Niches To Increase Hematopoietic Engraftment

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American Society of Hematology

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