Pediatric Blood & Marrow Transplant & Cellular Therapy

2025

Allogeneic haematopoietic stem-cell transplantation for children with refractory systemic juvenile idiopathic arthritis and associated lung disease: outcomes from an international, retrospective cohort study

Matt, M. G., Drozdov, D., Bendstrup, E., Glerup, M., Hauge, E. M., Masmas, T., Schneider, E. C., Zeilhofer, U. B., Abu-Arja, R. F., Driest, K. D., Oved, J. H., Onel, K., Ebens, C. L., Chellapandian, D., Chandrakasan, S., Prahalad, S., Roth, J., Prockop, S. E., Silva, J. & Schapiro, A. H. & 5 others, Towe, C., Chandra, S., Grom, A., Schulert, G. S. & Marsh, R. A., 2025, (Accepted/In press) In: The Lancet Rheumatology.

Research output: Contribution to journal › Article › peer-review

1 Scopus citations

Analysis of readability of the top web searches for pediatric inborn errors of fatty acid metabolism

Sawyer, K., Miller, W. C., Popp, C. M., Strege, C., Eide, C. R. & Tolar, J., Mar 2025, In: Molecular Genetics and Metabolism Reports. 42, 101195.

Research output: Contribution to journal › Article › peer-review

Open Access

Analysis of serum oligosaccharides by UPLC-MS/MS for diagnosis and treatment monitoring of patients with alpha-mannosidosis

Kubaski, F., Cason, A., Herbst, Z. M., Kharbanda, S., Lund, T. C., Orchard, P. J. & Pollard, L., Mar 2025, In: Molecular Genetics and Metabolism. 144, 3, 109042.

Research output: Contribution to journal › Article › peer-review

A Novel Mouse Model for Cerebral Inflammatory Demyelination in X-Linked Adrenoleukodystrophy: Insights into Pathogenesis and Potential Therapeutic Targets

Hashemi, E., Srivastava, I. N., Aguirre, A., Yoseph, E. T., Kaushal, E., Awani, A., Ryu, J. K., Akassoglou, K., Talebian, S., Chu, P., Pisani, L., Musolino, P., Steinman, L., Doyle, K., Robinson, W. H., Sharpe, O., Cayrol, R., Orchard, P. J., Lund, T. & Vogel, H. & 4 others, Lenail, M., Han, M. H., Bonkowsky, J. L. & Van Haren, K. P., Feb 2025, In: Annals of Neurology. 97, 2, p. 296-312 17 p.

Research output: Contribution to journal › Article › peer-review

A PSMA-Targeted Tri-Specific Killer Engager Enhances NK Cell Cytotoxicity against Prostate Cancer

Phung, S. K., Zorko, N. A., Soignier, Y., Waller, R. L., Shackelford, M., Walker, J. T., Nelson, T. D., Selleck, C., Bendzick, L. E., Kotz, L. E., Kile, Q. M., Bozicevich, A. J., Miller, S. E., Khaw, M., Shetty, M., Hinderlie, P., Ehrhardt, M., Li, Y., Luo, X. & Dehm, S. M. & 4 others, Antonarakis, E. S., Kennedy, P. R., Miller, J. S. & Felices, M., Feb 1 2025, In: Cancer Immunology Research. 13, 2, p. 258-272 15 p.

Research output: Contribution to journal › Article › peer-review

Bodyweight and Absolute Lymphocyte Count-Based Dosing of Rabbit Anti-thymocyte Globulin Results in Early CD4⁺ Immune Reconstitution in Patients with Inborn Errors of Metabolism Undergoing Umbilical Cord Blood Transplantation

Drozdov, D., Kandil, J., Long, S. E., Demorest, C. V., Cao, Q., Lund, T. C., Gupta, A. O., Boelens, J. J. & Orchard, P. J., 2025, (Accepted/In press) In: Transplantation and Cellular Therapy.

Research output: Contribution to journal › Article › peer-review

Open Access

Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series

Pillai, N. R., Liu, N., Li, X., Li, X., Ahrens-Nicklas, R., Adang, L., Eisengart, J. B., Bronken, G., Gupta, A., Lund, T. C., Whitley, C. B., Elsea, S. H. & Orchard, P. J., Dec 2025, In: Communications Medicine. 5, 1, 12.

Research output: Contribution to journal › Article › peer-review

Open Access

Characterization of gallbladder disease in metachromatic leukodystrophy across the lifespan

Mutua, S., Sevagamoorthy, A., Woidill, S., Orchard, P. J., Gavazzi, F., MacFarland, S. P., Russo, P., Vanderver, A. & Adang, L. A., Jan 2025, In: Molecular Genetics and Metabolism. 144, 1, 109003.

Research output: Contribution to journal › Article › peer-review

Different phenotypes with different endings—Telomere biology disorders and cancer predisposition with long telomeres

Team Telomere and the Clinical Care Consortium for Telomere-Associated Ailments (CCCTAA), Jan 2025, In: British journal of haematology. 206, 1, p. 69-73 5 p.

Research output: Contribution to journal › Article › peer-review

Open Access

Effect of Notch1 signaling on muscle engraftment and maturation from pluripotent stem cells

Yamashita, A. M. S., Garay, B. I., Kim, H., Bosnakovski, D., Abrahante, J. E., Azzag, K., Abreu, P., Ahlquist, A. & Perlingeiro, R. C. R., Feb 11 2025, In: Stem Cell Reports. 20, 2, 102396.

Research output: Contribution to journal › Article › peer-review

Open Access

Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing

Pandey, S., Gao, X. D., Krasnow, N. A., McElroy, A., Tao, Y. A., Duby, J. E., Steinbeck, B. J., McCreary, J., Pierce, S. E., Tolar, J., Meissner, T. B., Chaikof, E. L., Osborn, M. J. & Liu, D. R., Jan 2025, In: Nature Biomedical Engineering. 9, 1, p. 22-39 18 p., 627.

Research output: Contribution to journal › Article › peer-review

Open Access

27 Scopus citations

Incidence of bacterial blood stream infections in patients with acute GVHD

Wallis, W., Gulbis, A. M., Wang, T., Lee, C. J., Sharma, A., Williams, K. M., Nishihori, T., Prestidge, T., Gowda, L., Byrne, M., Krem, M. M., MacMillan, M. L., Kitko, C. L., Pidala, J., Spellman, S. R., Lee, S. J. & Alousi, A. M., Jan 2025, In: Bone marrow transplantation. 60, 1, p. 52-57 6 p.

Research output: Contribution to journal › Article › peer-review

1 Scopus citations

Insights From Minnesota on Newborn Screening for Adrenoleukodystrophy: A 5-Year Update

Rayannavar, A., Billington, C. J., Tryon, R., Kaye, T., Gupta, A., Lund, T. C., Lteif, A., Adriatico, K., Orchard, P. J., Miller, B. S. & Pillai, N. R., 2025, (Accepted/In press) In: American Journal of Medical Genetics, Part A.

Research output: Contribution to journal › Article › peer-review

Open Access

Prognostication and Biomarker Potential of C26:0 Lysophosphatidylcholine in Adrenoleukodystrophy

Billington, C. J., Rayannavar, A., Tryon, R., Kaye, T., Gupta, A. O., Lund, T. C., Lteif, A., Adriatico, K. L., Orchard, P. J., Miller, B. S. & Pillai, N. R., 2025, (Accepted/In press) In: JAMA Pediatrics.

Research output: Contribution to journal › Article › peer-review

Targeting cell-surface VISTA expression on allo-specific naïve T cells promotes tolerance

Koehn, B. H., Nowak, E. C., Skopelja-Gardner, S., Saha, A., Zaiken, M. C., Allred, J., Peng, Y., Davis, W. L., Le Mercier, I., Schwertner, N., Molloy, M. J., Rothstein, J., Carriere, C., Riddle, M. J., Eide, C. R., Tolar, J., Panoskaltsis-Mortari, A., Smith, K. D., Hippen, K. L. & Kim, T. K. & 2 others, Noelle, R. J. & Blazar, B. R., 2025, (Accepted/In press) In: Blood.

Research output: Contribution to journal › Article › peer-review

2024

Accelerated Aging and Microsatellite Instability in Recessive Dystrophic Epidermolysis Bullosa–Associated Cutaneous Squamous Cell Carcinoma

Lee, C. A. A., Wu, S., Chow, Y. T., Kofman, E., Williams, V., Riddle, M., Eide, C., Ebens, C. L., Frank, M. H., Tolar, J., Hook, K. P., AlDubayan, S. H. & Frank, N. Y., Jul 2024, In: Journal of Investigative Dermatology. 144, 7, p. 1534-1543.e2

Research output: Contribution to journal › Article › peer-review

1 Scopus citations

Alloengraftment without significant toxicity or GVHD in CD45 antibody-drug conjugate–conditioned Fanconi anemia mice

Saha, A., Palchaudhuri, R., Lanieri, L., Hyzy, S., Riddle, M. J., Panthera, J., Eide, C. R., Tolar, J., Panoskaltsis-Mortari, A., Gorfinkel, L., Tkachev, V., Gerdemann, U., Alvarez-Calderon, F., Palato, E. R., MacMillan, M. L., Wagner, J. E., Kean, L. S., Osborn, M. J., Kiem, H. P. & Scadden, D. T. & 2 others, Olson, L. M. & Blazar, B. R., May 23 2024, In: Blood. 143, 21, p. 2201-2216 16 p.

Research output: Contribution to journal › Article › peer-review

5 Scopus citations

Allogeneic Hematopoietic Cell Transplant For Bone Marrow Failure or Myelodysplastic Syndrome in Dyskeratosis Congenita/Telomere Biology Disorders: Single-Center, Single-Arm, Open-Label Trial of Reduced-Intensity Conditioning Without Radiation

Dimitrov, M., Merkle, S., Cao, Q., Tryon, R. K., Vercellotti, G. M., Holtan, S. G., Kao, R. L., Srikanthan, M., Terezakis, S. A., Tolar, J. & Ebens, C. L., Oct 2024, In: Transplantation and Cellular Therapy. 30, 10, p. 1005.e1-1005.e17

Research output: Contribution to journal › Article › peer-review

A model of lymphocryptovirus-associated post-transplant lymphoproliferative disorder in immunosuppressed Mauritian cynomolgus macaques

Wu, H. L., Weber, W. C., Waytashek, C. M., Boyle, C. D., Reed, J. S., Bateman, K. B., Fisher, H. K., Chen, Y., Armantrout, K., Swanson, T., Shriver-Munsch, C., Northrup, M., Fischer, M., Biswas, S., Templon, J., Panoskaltsis-Mortari, A., Burwitz, B. J., Johnson, A. L., Colgin, L. & Lewis, A. D. & 9 others, Smedley, J. V., Axthelm, M. K., Skalsky, R., Meyers, G., Maziarz, R. T., Mittra, E., Berg, M., Stanton, J. J. & Sacha, J. B., Nov 2024, In: PLoS pathogens. 20, 11, e1012644.

Research output: Contribution to journal › Article › peer-review

Open Access

1 Scopus citations

Amphiregulin, ST2, and REG3α biomarker risk algorithms as predictors of nonrelapse mortality in patients with acute GVHD

Etra, A., Jurdi, N. E., Katsivelos, N., Kwon, D., Gergoudis, S., Morales, G., Spyrou, N., Kowalyk, S., Aguayo-Hiraldo, P., Akahoshi, Y., Ayuk, F., Baez, J., Betts, B. C., Chanswangphuwana, C., Chen, Y. B., Choe, H., DeFilipp, Z., Gleich, S., Hexner, E. & Hogan, W. J. & 18 others, Holler, E., Kitko, C. L., Kraus, S., Malki, M. A., MacMillan, M., Pawarode, A., Quagliarella, F., Qayed, M., Reshef, R., Schechter, T., Vasova, I., Weisdorf, D., Wölfl, M., Young, R., Nakamura, R., Ferrara, J. L. M., Levine, J. E. & Holtan, S., Jun 25 2024, In: Blood Advances. 8, 12, p. 3284-3292 9 p.

Research output: Contribution to journal › Article › peer-review

Open Access

Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment

Keller, M. D., Hanley, P. J., Chi, Y. Y., Aguayo-Hiraldo, P., Dvorak, C. C., Verneris, M. R., Kohn, D. B., Pai, S. Y., Dávila Saldaña, B. J., Hanisch, B., Quigg, T. C., Adams, R. H., Dahlberg, A., Chandrakasan, S., Hasan, H., Malvar, J., Jensen-Wachspress, M. A., Lazarski, C. A., Sani, G. & Idso, J. M. & 29 others, Lang, H., Chansky, P., McCann, C. D., Tanna, J., Abraham, A. A., Webb, J. L., Shibli, A., Keating, A. K., Satwani, P., Muranski, P., Hall, E., Eckrich, M. J., Shereck, E., Miller, H., Mamcarz, E., Agarwal, R., De Oliveira, S. N., Vander Lugt, M. T., Ebens, C. L., Aquino, V. M., Bednarski, J. J., Chu, J., Parikh, S., Whangbo, J., Lionakis, M., Zambidis, E. T., Gourdine, E., Bollard, C. M. & Pulsipher, M. A., Dec 2024, In: Nature communications. 15, 1, 3258.

Research output: Contribution to journal › Article › peer-review

Open Access

4 Scopus citations

Association of busulfan exposure and outcomes after HCT for patients with an inborn error of immunity

Bognàr, T., Garcia-Rosa, M., Lalmohamed, A., Güngör, T., Hauri-Hohl, M., Prockop, S., Oram, L., Pai, S. Y., Brooks, J., Savic, R. M., Dvorak, C. C., Long-Boyle, J. R., Krajinovic, M., Bittencourt, H., Teyssier, A. C., Théoret, Y., Martinez, C., Egberts, T. C. G., Morales, E. & Slatter, M. & 18 others, Cuvelier, G. D. E., Chiesa, R., Wynn, R. F., Coussons, M., Cicalese, M. P., Ansari, M., Long, S. E., Ebens, C. L., Lust, H., Chaudhury, S., Nath, C. E., Shaw, P. J., Keogh, S. J., van der Stoep, M. Y. C. E., Bredius, R., Lindemans, C. A., Boelens, J. J. & Bartelink, I. H., Oct 8 2024, In: Blood Advances. 8, 19, p. 5137-5145 9 p.

Research output: Contribution to journal › Article › peer-review

Open Access

2 Scopus citations

Audacity of gene therapy

Dimitrov, M., Ebens, C. L. & Tolar, J., Dec 2024, In: British Journal of Dermatology. 191, 6, p. 1009-1011 3 p.

Research output: Contribution to journal › Article › peer-review

Biochemical signatures of disease severity in multiple sulfatase deficiency

Adang, L. A., Mowafy, S., Herbst, Z. M., Zhou, Z., Schlotawa, L., Radhakrishnan, K., Bentley, B., Pham, V., Yu, E., Pillai, N. R., Orchard, P. J., De Castro, M., Vanderver, A., Pasquali, M., Gelb, M. H. & Ahrens-Nicklas, R. C., Mar 2024, In: Journal of Inherited Metabolic Disease. 47, 2, p. 374-386 13 p.

Research output: Contribution to journal › Article › peer-review

6 Scopus citations

Bone marrow transplantation increases sulfatase activity in somatic tissues in a multiple sulfatase deficiency mouse model

Presa, M., Pham, V., Ray, S., Piec, P. A., Ryan, J., Billings, T., Coombs, H., Schlotawa, L., Lund, T., Ahrens-Nicklas, R. C. & Lutz, C., Dec 2024, In: Communications Medicine. 4, 1, 215.

Research output: Contribution to journal › Article › peer-review

Open Access

Cardiac Development Long Non-Coding RNA (CARDEL) Is Activated during Human Heart Development and Contributes to Cardiac Specification and Homeostasis

Pereira, I. T., Gomes-Júnior, R., Hansel-Frose, A., França, R. S. V., Liu, M., Soliman, H. A. N., Chan, S. S. K., Dudley, S. C., Kyba, M. & Dallagiovanna, B., Jun 2024, In: Cells. 13, 12, 1050.

Research output: Contribution to journal › Article › peer-review

Open Access

Chondroitin sulfate proteoglycan 4 increases invasion of recessive dystrophic epidermolysis bullosa-associated cutaneous squamous cell carcinoma by modifying transforming growth factor-β signalling

Macaulay, A. R. K., Yang, J., Price, M. A., Forster, C. L., Riddle, M. J., Ebens, C. L., Albert, F. W., Giubellino, A., Mc Carthy, J. B. & Tolar, J., Dec 23 2024, In: British Journal of Dermatology. 192, 1, p. 104-117 14 p.

Research output: Contribution to journal › Article › peer-review

1 Scopus citations

Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice

Smith, M. C., Belur, L. R., Karlen, A. D., Erlanson, O., Furcich, J., Lund, T. C., Seelig, D., Kitto, K. F., Fairbanks, C. A., Kim, K. H., Buss, N. & McIvor, R. S., Mar 14 2024, In: Molecular Therapy Methods and Clinical Development. 32, 1, 101201.

Research output: Contribution to journal › Article › peer-review

Open Access

5 Scopus citations

Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States

Adang, L. A., Bonkowsky, J. L., Boelens, J. J., Mallack, E., Ahrens-Nicklas, R., Bernat, J. A., Bley, A., Burton, B., Darling, A., Eichler, F., Eklund, E., Emrick, L., Escolar, M., Fatemi, A., Fraser, J. L., Gaviglio, A., Keller, S., Patterson, M. C., Orchard, P. & Orthmann-Murphy, J. & 12 others, Santoro, J. D., Schöls, L., Sevin, C., Srivastava, I. N., Rajan, D., Rubin, J. P., Van Haren, K., Wasserstein, M., Zerem, A., Fumagalli, F., Laugwitz, L. & Vanderver, A., Jul 2024, In: Cytotherapy. 26, 7, p. 739-748 10 p.

Research output: Contribution to journal › Article › peer-review

5 Scopus citations

Creation and characterization of novel rat model for recessive dystrophic epidermolysis bullosa: Frameshift mutation of the Col7a1 gene leads to severe blistered phenotype

Stone, W., Strege, C., Miller, W., Geurts, A. M., Grzybowski, M., Riddle, M., Lees, C., Eide, C., Keene, D. R., Tufa, S. F., Seelig, D., McGrath, J. & Tolar, J., May 2024, In: PloS one. 19, 5 May, e0302991.

Research output: Contribution to journal › Article › peer-review

Open Access

Developmental delay can precede neurologic regression in early onset metachromatic leukodystrophy

Adang, L. A., Groeschel, S., Grzyb, C., D'Aiello, R., Gavazzi, F., Sherbini, O., Bronner, N., Patel, A., Vincent, A., Sevagamoorthy, A., Mutua, S., Muirhead, K., Schmidt, J., Pizzino, A., Yu, E., Jin, D., Eichler, F., Fraser, J. L., Emrick, L. & Van Haren, K. & 28 others, Boulanger, J. M., Ruzhnikov, M., Sylvain, M., Nguyen, C. T. É., Potic, A., Keller, S., Fatemi, A., Uebergang, E., Poe, M., Yazdani, P. A., Bernat, J., Lindstrom, K., Bonkowsky, J. L., Bernard, G., Stutterd, C. A., Orchard, P., Gupta, A. O., Ljungberg, M., Groenborg, S., Zambon, A., Locatelli, S., Fumagalli, F., Elguen, S., Kehrer, C., Krägeloh-Mann, I., Shults, J., Vanderver, A. & Escolar, M. L., Aug 2024, In: Molecular Genetics and Metabolism. 142, 4, 108521.

Research output: Contribution to journal › Article › peer-review

Development of an Anatomically Accurate Three-Dimensional Simulation Model for Pediatric Central Line Placement

Carver, A., Bjorklund, A., Broomhead, J., Graba, E., Prabhu, S. & Fischer, G., Mar 1 2024, In: Journal of Medical Devices, Transactions of the ASME. 18, 1, 011004.

Research output: Contribution to journal › Article › peer-review

Development of specific growth charts for children with Fanconi anemia

Barbus, C., Rayannavar, A., Miller, B. S., Jenkins, M. J., Addo, O. Y., Rayes, A., Ahrweiler, N., Olson, A., Pohlkamp, Z., Wagner, J. E. & MacMillan, M. L., Jul 2024, In: American Journal of Medical Genetics, Part A. 194, 7, e63554.

Research output: Contribution to journal › Article › peer-review

Open Access

2 Scopus citations

Diffusion Tensor Imaging in Boys with Adrenoleukodystrophy: Identification of Cerebral Disease and Association with Neurocognitive Outcomes

Pierpont, E. I., Labounek, R., Gupta, A., Lund, T., Orchard, P. J., Dobyns, W. B., Bondy, M., Paulson, A., Metz, A., Shanley, R., Wozniak, J. R., Mueller, B. A., Loes, D., Nascene, D. & Nestrasil, I., Aug 16 2024, In: Neurology. 103, 5, e209764.

Research output: Contribution to journal › Article › peer-review

Estradiol deficiency as a consequence of aging contributes to the depletion of the satellite cell pool in female mice

Sullivan, B. P., Larson, A. A., Shams, A. S., McMillin, S. L., Ebeling, M. C., Peng, S., Kyba, M. & Lowe, D. A., 2024, (Accepted/In press) In: Aging cell.

Research output: Contribution to journal › Article › peer-review

FANCA c.3624C>T (p.Ser1208=) is a hypomorphic splice variant associated with delayed onset of Fanconi anemia

Ramanagoudr-Bhojappa, R., Tryon, R., Lach, F. P., Donovan, F. X., Maxwell, R., Rosenberg, A., MacMillan, M. L., Wagner, J. E., Auerbach, A. D., Smogorzewska, A. & Chandrasekharappa, S. C., Feb 27 2024, In: Blood Advances. 8, 4, p. 899-908 10 p.

Research output: Contribution to journal › Article › peer-review

Open Access

Fanconi anemia neuroinflammatory syndrome: brain lesions and neurologic injury in Fanconi anemia

Bartlett, A. L., Wagner, J. E., Jones, B., Wells, S., Sabulski, A., Fuller, C. & Davies, S. M., Jun 25 2024, In: Blood Advances. 8, 12, p. 3027-3037 11 p.

Research output: Contribution to journal › Article › peer-review

Open Access

1 Scopus citations

Healthcare cost and utilization for chimeric antigen receptor (CAR) T-cell therapy in the treatment of pediatric acute lymphoblastic leukemia: A commercial insurance claims database analysis

Hoover, A., Reimche, P., Watson, D., Tanner, L., Gilchrist, L., Finch, M., Messinger, Y. H. & Turcotte, L. M., Feb 2024, In: Cancer Reports. 7, 2, e1980.

Research output: Contribution to journal › Article › peer-review

Open Access

2 Scopus citations

Hemangiosarcoma Cells Promote Conserved Host-derived Hematopoietic Expansion

Kim, J. H., Schulte, A. J., Sarver, A. L., Lee, D., Angelos, M. G., Frantz, A. M., Forster, C. L., O’Brien, T. D., Cornax, I., O’Sullivan, M. G., Cheng, N., Lewellen, M., Oseth, L. A., Kumar, S., Bullman, S., Pedamallu, C. S., Goyal, S. M., Meyerson, M., Lund, T. C. & Breen, M. & 4 others, Lindblad-Toh, K., Dickerson, E. B., Kaufman, D. S. & Modiano, J. F., Jun 2024, In: Cancer Research Communications. 4, 6, p. 1467-1480 14 p.

Research output: Contribution to journal › Article › peer-review

Open Access

4 Scopus citations

Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy

Duncan, C. N., Bledsoe, J. R., Grzywacz, B. J., Beckman, A., Bonner, M., Eichler, F. S., Kühl, J. S., Harris, M. H., Slauson, S., Colvin, R. A., Prasad, V. K., Downey, G. F., Pierciey, F. J., Kinney, M. A., Foos, M., Lodaya, A., Floro, N., Parsons, G., Dietz, A. C. & Gupta, A. O. & 3 others, Orchard, P. J., Thakar, H. L. & Williams, D. A., Oct 10 2024, In: New England Journal of Medicine. 391, 14, p. 1287-1301 15 p.

Research output: Contribution to journal › Article › peer-review

17 Scopus citations

Impact of the COVID-19 pandemic on utilization and cost for care of pediatric and young adult ALL

Hoover, A., Watson, D., Reimche, P., Tanner, L., Gilchrist, L., Finch, M., Messinger, Y. H. & Turcotte, L. M., Dec 2024, In: BMC Research Notes. 17, 1, 112.

Research output: Contribution to journal › Article › peer-review

Open Access

Inducing positive inotropy in human iPSC-derived cardiac muscle by gene editing-based activation of the cardiac α-myosin heavy chain

Bedada, F. B., Thompson, B. R., Mikkila, J. L., Chan, S. S. K., Choi, S. H., Toso, E. A., Kyba, M. & Metzger, J. M., Dec 2024, In: Scientific reports. 14, 1, 3915.

Research output: Contribution to journal › Article › peer-review

Open Access

1 Scopus citations

Integrating Porous Silicon Nanoneedles within Medical Devices for Nucleic Acid Nanoinjection

Wang, C., Gu, C., Popp, C., Vashisth, P., Mustfa, S. A., Martella, D. A., Spiteri, C., McLennan, S., Sun, N., Riddle, M., Eide, C. R., Parsons, M., Tolar, J., McGrath, J. A. & Chiappini, C., Jun 11 2024, In: ACS nano. 18, 23, p. 14938-14953 16 p.

Research output: Contribution to journal › Article › peer-review

Open Access

6 Scopus citations

International Society for Cell & Gene Therapy Stem Cell Engineering Committee report on the current state of hematopoietic stem and progenitor cell–based genomic therapies and the challenges faced

Gupta, A. O., Azul, M., Bhoopalan, S. V., Abraham, A., Bertaina, A., Bidgoli, A., Bonfim, C., DeZern, A., Li, J., Louis, C. U., Purtill, D., Ruggeri, A., Boelens, J. J., Prockop, S. & Sharma, A., Nov 2024, In: Cytotherapy. 26, 11, p. 1411-1420 10 p.

Research output: Contribution to journal › Article › peer-review

2 Scopus citations

Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I

Belur, L. R., Huber, A. K., Mantone, H., Robertson, M., Smith, M. C., Karlen, A. D., Kitto, K. F., Ou, L., Whitley, C. B., Braunlin, E., Furcich, J., Lund, T. C., Seelig, D., Fairbanks, C. A., Buss, N., Kim, K. H. & McIvor, R. S., Dec 12 2024, In: Molecular Therapy Methods and Clinical Development. 32, 4, 101369.

Research output: Contribution to journal › Article › peer-review

Open Access

In vivo PSC differentiation as a platform to identify factors for improving the engraftability of cultured muscle stem cells

Xie, N., Robinson, K., Sundquist, T. & Chan, S. S. K., 2024, In: Frontiers in Cell and Developmental Biology. 12, 1362671.

Research output: Contribution to journal › Article › peer-review

Open Access

Lactate supports Treg function and immune balance via MGAT1 effects on N-glycosylation in the mitochondria

Zhou, J., Gu, J., Qian, Q., Zhang, Y., Huang, T., Li, X., Liu, Z., Shao, Q., Liang, Y., Qiao, L., Xu, X., Chen, Q., Xu, Z., Li, Y., Gao, J., Pan, Y., Wang, Y., O'Connor, R., Hippen, K. L. & Lu, L. & 1 others, Blazar, B. R., Oct 15 2024, In: Journal of Clinical Investigation. 134, 20, e175897.

Research output: Contribution to journal › Article › peer-review

Open Access

2 Scopus citations

Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy

Eichler, F., Duncan, C. N., Musolino, P. L., Lund, T. C., Gupta, A. O., De Oliveira, S., Thrasher, A. J., Aubourg, P., Kühl, J. S., Loes, D. J., Amartino, H., Smith, N., Folloni Fernandes, J., Sevin, C., Sankar, R., Hussain, S. A., Gissen, P., Dalle, J. H., Platzbecker, U. & Downey, G. F. & 6 others, McNeil, E., Demopoulos, L., Dietz, A. C., Thakar, H. L., Orchard, P. J. & Williams, D. A., Oct 10 2024, In: New England Journal of Medicine. 391, 14, p. 1302-1312 11 p.

Research output: Contribution to journal › Article › peer-review

7 Scopus citations

Lipidomic biomarkers in plasma correlate with disease severity in adrenoleukodystrophy

Jaspers, Y. R. J., Yska, H. A. F., Bergner, C. G., Dijkstra, I. M. E., Huffnagel, I. C., Voermans, M. M. C., Wever, E., Salomons, G. S., Vaz, F. M., Jongejan, A., Hermans, J., Tryon, R. K., Lund, T. C., Köhler, W., Engelen, M. & Kemp, S., Dec 2024, In: Communications Medicine. 4, 1, 175.

Research output: Contribution to journal › Article › peer-review

Open Access

6 Scopus citations

Liver disease and transplantation in telomere biology disorders: An international multicenter cohort

Clinical Care Consortium for Telomere-associated Ailments (CCCTAA), Jun 2024, In: Hepatology Communications. 8, 7, p. e0462

Research output: Contribution to journal › Article › peer-review

Open Access

1 Scopus citations

Pediatric Blood & Marrow Transplant & Cellular Therapy

Research output

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