Research Summary

Current Non-NIH Awards:
TR3-05535 Cowan (PI) 11/01/2012-10/31/2015
California Institute of Regenerative Medicine vGene Correction of Autologous Hematopoietic Stem Cells in Artemis Deficient SCID
Goal is to complete the preclinical testing necessary for a clinical gene therapy trial in children with Artemis-SCID. The focus is on optimizing non-chemotherapy approaches to opening marrow niches, studying potential genotoxicity, and demonstrating that gene transduced Artemis deficient hematopoietic stem cells mature in vitro and in vivo into T and B cells.
Role: PI

DR2A-05365 Shizuru (PI) 07/01/2013-06/30/2017
California Institute of Regenerative Medicine vA monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants
Goal is to bring to a clinical trial a monoclonal antibody that targets hematopoietic stem cells. The antibody will be evaluated in a non-human primate model followed by a prospective phase I/II clinical trial in children with SCID.
Role: Co-PI

Overview:
I am a pediatric immunologist, Professor of Pediatrics and Chief, Allergy Immunology and BMT Division at UCSF. I led the planning for what ultimately became the Primary Immune Deficiency Treatment Consortium (PIDTC), an NIH-funded group of 43 centers in the US and Canada focused on PID in which I am the PI and UCSF is the lead institution since 2009. I am on the Steering and Executive Committees of the Pediatric Blood and Marrow Transplant Consortium (PBMTC) and established and led for three years the Pediatric Special Interest Group within the American Society of Blood and Marrow Transplantation. My career has focused on children with PID, with over 180 peer-reviewed publications. My major research interests include SCID in Athabascan-speaking Native Americans (SCIDA), gene therapy for children with Artemis-deficient SCID, non-chemotherapy approaches to opening marrow niches, and the use of haploidentical donors and reduced-toxicity transplantation for childhood diseases. I performed one of the initial haplocompatible T cell depleted marrow transplants for SCID in North America in 1982 and reported one of the first in utero transplants in the world, the first patient with multiple biotin-dependent carboxylase deficiencies and immune deficiency, and the first children with HIV infection. I have treated the largest cohort of children with SCIDA and the largest number of babies diagnosed through newborn screening with SCID in North America, mapped the genetic locus of the gene for SCIDA and radiation sensitive SCID (RS-SCID), and identified the mutation in DCLRE1C responsible for SCIDA in Navajo and Apache children. I designed and completed one of the few prospective single institution trials of haplocompatible HCT for children with SCID and initiated and completed the first large study of children with SCID transplanted in North America and reported by the PIDTC.

Research Funding

  • March 2, 2011 - February 28, 2026 - Annual Primary Immune Deficiency Treatment Consortium (PIDTC) Workshop and Education Day , Co-Principal Investigator . Sponsor: NIH, Sponsor Award ID: R13AI094943
  • September 12, 2009 - August 31, 2024 - Primary Immune Deficiency Treatment Consortium , Principal Investigator . Sponsor: NIH, Sponsor Award ID: U54AI082973
  • May 1, 2018 - April 30, 2023 - Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells , Principle Investigator . Sponsor: California Institute of Regenerative Medicine, Sponsor Award ID: CLIN2-10830
  • April 1, 2017 - March 31, 2022 - Lentiviral Gene Therapy for Infants with X-linked Severe Combined Immunodeficiency using Autologous Bone Marrow Stem Cells and Busulfan Conditioning , Co-PI . Sponsor: California Institute of Regenerative Medicine, Sponsor Award ID: CLIN2-09504
  • March 1, 1982 - May 31, 2015 - Bio-Organic Biomedical Mass Spectrometry Resource , Co-Investigator . Sponsor: NIH, Sponsor Award ID: P41RR001614
  • June 1, 1975 - April 30, 2015 - California National Primate Research Center , Co-Investigator . Sponsor: NIH, Sponsor Award ID: P51RR000169
  • December 1, 1974 - March 31, 2009 - General Clinical Research Center , Co-Investigator . Sponsor: NIH, Sponsor Award ID: M01RR000079
  • December 1, 1981 - March 31, 2007 - PEDIATRIC CLINICAL RESEARCH CENTER , Co-Investigator . Sponsor: NIH, Sponsor Award ID: M01RR001271
  • January 1, 1998 - March 31, 2006 - Tolerance post in utero transplantation , Principal Investigator . Sponsor: NIH, Sponsor Award ID: R01HL058842
  • September 30, 1993 - June 30, 2003 - GENE LINKAGE STUDY OF IMMUNODEFICIENCY IN NAVAJO INDIANS , Principal Investigator . Sponsor: NIH, Sponsor Award ID: R01AI028339
  • August 1, 1990 - July 31, 1995 - INDUCTION OF TOLERANCE IN MARROW TRANSPLANTATION , Principal Investigator . Sponsor: NIH, Sponsor Award ID: P01AI029512
  • September 30, 1986 - March 31, 1993 - HIV INFECTION IN CHILDREN OF IV DRUG ABUSING WOMEN , Principal Investigator . Sponsor: NIH, Sponsor Award ID: R01DA004331

Education

Massachusetts Institute of Technology, Cambridge, MA, B.S., 1966, Electrical Engineering
University of Pennsylvania, Philadelphia, PA, M.D., 1970, Medicine
University of California, San Francisco, Intern/Resident, 1975-77, Pediatrics
University of California, San Francisco, Fellow, 1977-79, Immunology

Honors & Awards

  • 1966
    Tau Beta Pi, Engineer Honor Society
  • 1968-70
    Charles Hovies Scholarship
  • 1979-82
    Pediatric Clinical Research Center Investigator Award

Selected Publications

  1. Winestone LE, Logan BR, Liu X, Williams O, Dunn EA, Thakar MS, Kidd SA, Mousallem T, Dorsey MJ, O'Reilly RJ, Kapoor N, Forbes Satter L, Kapadia M, Jyonouchi SC, Chandra S, Ebens CL, Chellapandian D, Chaudhury S, Chen K, Dávila Saldaña BJ, Rayes A, Quigg TC, Chandrakasan S, Bednarski JJ, DeSantes KB, Teira P, Gillio AP, Eissa H, Knutsen AP, Aquino VM, Shereck EB, Moore TB, Caywood EH, Vander Lugt MT, Rozmus J, Broglie L, Yu LC, Shah AJ, Joshi AY, Buckley RH, Dara J, Oved JH, Abdel Azim H, Martinez CA, Prockop S, Sullivan KE, Bleesing JJ, Keller MD, Madden LM, Parikh S, Shenoy S, Seroogy CM, Rubin T, Licata J, Andolina JR, Kohn DB, Burroughs L, Leiding JW, Marsh RA, Notarangelo LD, Pai SY, Torgerson TR, Pulsipher MA, Heimall J, Cuvelier GDE, Griffith LM, Puck JM, Dvorak CC, Cowan MJ, Haddad E. Newborn screening reduces survival disparities in SCID after stem cell transplant: A PIDTC report. J Hum Immun. 2026 Sep 07; 2(5):e20250231. View on PubMed
  2. Arnold DE, Eissa H, Dunn E, Cuvelier GDE, Wright NAM, Marsh RA, Parikh S, Saldana BD, Vander Lugt MT, Bacchetta R, Satter LF, Chandrakasan S, Kellner E, Allenspach EJ, Schmitt EG, Logan B, Burroughs L, Cowan MJ, Griffith LM, Notarangelo L, Pai SY, Pulsipher MA, Dvorak CC, Haddad E, Puck JM, Torgerson TR, Leiding JW, Heimall J, Chan AY. IMPACT (Immune Monitoring and Phenotype Assessment of Clinical Trajectory) for Patients With Inborn Errors of Immunity From the Primary Immune Deficiency Treatment Consortium (PIDTC). J Allergy Clin Immunol Pract. 2026 Jun 03. View on PubMed
  3. Alexander JL, Dávila Saldaña BJ, Brazauskas R, Dammalapati SG, Griffith LM, Shah AJ, Shimano KA, Ochs HD, Bleesing JJ, Ebens CL, Kapadia M, Bauchat A, Kapoor N, Oved JH, Eissa H, Lust H, Keller MD, Haines H, Chandrakasan S, Talano JA, Rayes A, Madden L, Shereck E, Miller HK, Satter LF, Martinez C, Rozmus J, Bednarski JJ, Yu LC, Chellapandian D, Aquino VM, Knutsen A, Chong H, Chopek A, Gillio AP, Joshi A, Rangarajan H, Moore TB, Andolina JR, DeSantes KB, Vander Lugt M, Prockop SE, Shyr DC, Sullivan KE, Parikh S, Weinacht KG, Torgerson TR, Marsh R, Dvorak CC, Chan AY, Haddad E, Heimall JR, Pulsipher MA, Leiding JW, Kohn DB, Puck JM, Notarangelo LD, Rawlings DJ, Cowan MJ, Petrovic A, Pai SY, Burroughs LM. Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report. Blood Adv. 2026 Mar 10; 10(5):1783-1798. View on PubMed
  4. Rayes A, Logan BR, Liu X, Dara J, Buckley RH, Oved JH, Kapoor N, Kapadia M, Chandra S, Martinez CA, Bunin NJ, Chandrakasan S, Chen K, Bednarski JJ, Haines HL, Eissa H, Talano JM, Keller MD, Ebens CL, Chaudhury S, Shereck EB, Aquino VM, Knutsen AP, Alexander JL, Gillio AP, Chellapandian D, Shah AJ, Miller HK, Vander Lugt MT, Seroogy CM, Dorsey MJ, Mousallem T, Parrott RE, O'Reilly RJ, Aguayo-Hiraldo PI, Prockop SE, Dávila Saldaña BJ, Thakar MS, Burroughs LM, Torgerson TR, Leiding JW, Marsh RA, Griffith LM, Pulsipher MA, Kohn DB, Notarangelo L, Cowan MJ, Puck JM, Cuvelier GDE, Heimall J, Haddad E, Pai SY, Dvorak CC. Outcomes following matched sibling donor transplantation for severe combined immunodeficiency: a report from the PIDTC. Blood Adv. 2026 Feb 10; 10(3):887-900. View on PubMed
  5. Marsh RA, Cowan MJ. The evolving landscape of primary hemophagocytic lymphohistiocytosis: Long-term outcomes following hematopoietic cell transplantation. J Allergy Clin Immunol. 2026 Feb; 157(2):340-341. View on PubMed
  6. Chandrakasan S, Westbrook A, Griffith LM, Hagin D, Iyengar S, Mangurian C, Scalchunes C, Sullivan KE, Zablocki A, Bakshi N, Sinha C, Burroughs LM, Chan AY, Dvorak CC, Haddad E, Heimall J, Kohn DB, Leiding JW, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Marsh RA, Torgerson T, Cowan MJ, Ochs HD, Parikh S. Clinical spectrum of Wiskott-Aldrich syndrome carriers: Self-reported survey of 193 carriers. Clin Immunol. 2026 Feb; 283:110658. View on PubMed
  7. Brumm VL, Kidd SA, Logan BR, Chunara F, Heimall J, Griffith LM, Kohn DB, Sanchez L, Bednarski JJ, Martinez C, Lugt MV, Kapoor N, Wright N, Spitzer B, Oved JH, Chandra S, Chellapandian D, Ebens CL, Petrovic A, Rayes A, Haines HL, Lust H, Schofield HT, Christopher L, Harris LL, Satter LF, Burroughs L, Dvorak CC, Haddad E, Leiding JW, Marsh RA, Notarangelo LD, Pai SY, Pulsipher MA, Puck JM, Cowan MJ, Shah AJ. Neurodevelopmental outcomes following hematopoietic cell transplantation for patients with severe combined immunodeficiency (SCID): A PIDTC study. J Hum Immun. 2026 Jan 05; 2(1):e20250163. View on PubMed
  8. Jacovas VC, Zelnick M, McNulty S, Ross JE, Khurana N, Pan X, Nieto A, Martin S, McLean B, Elnagheeb MA, Cowan MJ, Puck JM, Hershfield MS, Verbsky J, Walter J, Allenspach EJ, Chan AY, van Oers NSC, Ghosh R, Piazza M, Yuan B, Notarangelo LD, Johnson BA, Chinn IK, Severe Combined Immunodeficiency Variant Curation Expert Panel. The ClinGen Severe Combined Immunodeficiency Disease Variant Curation Expert Panel: Specifications for classification of variants in ADA, DCLRE1C, IL2RG, IL7R, JAK3, RAG1, and RAG2. Genet Med. 2026 Jan; 28(1):101613. View on PubMed
  9. Arnold DE, Leiding JW, Logan B, Marsh RA, Griffith LM, Grunebaum E, Murguía-Favela L, Mallhi K, Chellapandian D, Deal CL, Lim SS, Prasad V, Heimall J, Chandrakasan S, Chen K, Yu LC, Seroogy CM, Gillio A, Bednarski JJ, Kapoor N, Moore TB, Cuvelier GDE, Touzot F, Rayes A, Ebens CL, Schaefer E, Bauchat A, Chopek A, Burroughs L, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson T, Malech HL, Kang EM, Parikh S. Umbilical Cord Blood Transplantation Provides an Alternative for Patients With Chronic Granulomatous Disease Lacking HLA-Matched Donors: A PIDTC Report. Transplant Cell Ther. 2026 Jan; 32(1):89.e1-89.e10. View on PubMed
  10. Vasandan AB, Abdelsamed HA, Boi SK, Ward G, Alli S, Mi T, Lan X, Zhang X, Cowan MJ, Puck JM, Petrovic A, Rawlings D, Zhou A, Mamcarz E, Gottschalk S, Zebley CC, Youngblood B. Innate-like memory T cells rapidly emerge in humans after gene therapy for SCID-X1. Immunity. 2025 Aug 12; 58(8):1922-1930.e3. View on PubMed
  11. Jacovas VC, Zelnick M, McNulty S, Ross JE, Khurana N, Pan X, Nieto A, Martin S, McLean B, Elnagheeb MA, Cowan MJ, Puck JM, Hershfield M, Verbsky J, Walter J, Allenspach E, Chan AY, van Oers NSC, Ghosh R, Piazza M, Yuan B, Notarangelo LD, Johnson BA, Chinn IK. The ClinGen Severe Combined Immunodeficiency Disease Variant Curation Expert Panel: Specifications for classification of variants in ADA , DCLRE1C , IL2RG , IL7R , JAK3 , RAG1 , and RAG2. medRxiv. 2025 Feb 13. View on PubMed
  12. Dvorak CC, Temple WC, Cisneros GS, Chu J, Winestone LE, Higham CS, Shimano KA, Kharbanda S, Avagyan S, Pauerstein P, Huang JN, Cheng G, Waters E, Winger BA, Cowan MJ, Long-Boyle JR. Younger children with nonmalignant disease have increased incidence of mixed myeloid chimerism after allogeneic hematopoietic cell transplantation with busulfan-based conditioning. Transplant Cell Ther. 2025 Apr; 31(4):259.e1-259.e15. View on PubMed
  13. Eissa H, Cowan MJ, Heimall J. Late effects following hematopoietic cell transplantation for severe combined immunodeficiency: critical factors and therapeutic options. Expert Rev Clin Immunol. 2025 01; 21(1):73-82. View on PubMed
  14. Miranda MA, Tsalatsanis A, Trotter JR, Arnold DE, Squire JD, Kidd S, Parikh S, Marsh RA, Griffith LM, Mallhi K, Chellapandian D, Lim SS, Grunebaum E, Sullivan KE, Newburger PE, Dinauer MC, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM, Morton FB, Leiding JW. High symptom burden in female X-linked chronic granulomatous disease carriers. Clin Immunol. 2024 Nov; 268:110364. View on PubMed
  15. Abraham RS, Basu A, Heimall JR, Dunn E, Yip A, Kapadia M, Kapoor N, Satter LF, Buckley R, O'Reilly R, Cuvelier GDE, Chandra S, Bednarski J, Chaudhury S, Moore TB, Haines H, Dávila Saldaña BJ, Chellapandian D, Rayes A, Chen K, Caywood E, Chandrakasan S, Lugt MTV, Ebens C, Teira P, Shereck E, Miller H, Aquino V, Eissa H, Yu LC, Gillio A, Madden L, Knutsen A, Shah AJ, DeSantes K, Barnum J, Broglie L, Joshi AY, Kleiner G, Dara J, Prockop S, Martinez C, Mousallem T, Oved J, Burroughs L, Marsh R, Torgerson TR, Leiding JW, Pai SY, Kohn DB, Pulsipher MA, Griffith LM, Notarangelo LD, Cowan MJ, Puck J, Dvorak CC, Haddad E. Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia. Clin Immunol. 2024 04; 261:109942. View on PubMed
  16. Grunebaum E, Arnold DE, Logan B, Parikh S, Marsh RA, Griffith LM, Mallhi K, Chellapandian D, Lim SS, Deal CL, Kapoor N, Murguía-Favela L, Falcone EL, Prasad VK, Touzot F, Bleesing JJ, Chandrakasan S, Heimall JR, Bednarski JJ, Broglie LA, Chong HJ, Kapadia M, Prockop S, Dávila Saldaña BJ, Schaefer E, Bauchat AL, Teira P, Chandra S, Parta M, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM, Leiding JW. Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A Primary Immune Deficiency Treatment Consortium study. J Allergy Clin Immunol. 2024 May; 153(5):1423-1431.e2. View on PubMed
  17. Leiding JW, Arnold DE, Parikh S, Logan B, Marsh RA, Griffith LM, Wu R, Kidd S, Mallhi K, Chellapandian D, Si Lim SJ, Grunebaum E, Falcone EL, Murguia-Favela L, Grossman D, Prasad VK, Heimall JR, Touzot F, Burroughs LM, Bleesing J, Kapoor N, Dara J, Williams O, Kapadia M, Oshrine BR, Bednarski JJ, Rayes A, Chong H, Cuvelier GDE, Forbes Satter LR, Martinez C, Vander Lugt MT, Yu LC, Chandrakasan S, Joshi A, Prockop SE, Dávila Saldaña BJ, Aquino V, Broglie LA, Ebens CL, Madden LM, DeSantes K, Milner J, Rangarajan HG, Shah AJ, Gillio AP, Knutsen AP, Miller HK, Moore TB, Graham P, Bauchat A, Bunin NJ, Teira P, Petrovic A, Chandra S, Abdel-Azim H, Dorsey MJ, Birbrayer O, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM. Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD. Blood. 2023 12 14; 142(24):2105-2118. View on PubMed
  18. Yan KK, Condori J, Ma Z, Metais JY, Ju B, Ding L, Dhungana Y, Palmer LE, Langfitt DM, Ferrara F, Throm R, Shi H, Risch I, Bhatara S, Shaner B, Lockey TD, Talleur AC, Easton J, Meagher MM, Puck JM, Cowan MJ, Zhou S, Mamcarz E, Gottschalk S, Yu J. Integrome signatures of lentiviral gene therapy for SCID-X1 patients. Sci Adv. 2023 10 06; 9(40):eadg9959. View on PubMed
  19. Eissa H, Thakar MS, Shah AJ, Logan BR, Griffith LM, Dong H, Parrott RE, O'Reilly RJ, Dara J, Kapoor N, Forbes Satter L, Chandra S, Kapadia M, Chandrakasan S, Knutsen A, Jyonouchi SC, Molinari L, Rayes A, Ebens CL, Teira P, Dávila Saldaña BJ, Burroughs LM, Chaudhury S, Chellapandian D, Gillio AP, Goldman F, Malech HL, DeSantes K, Cuvelier GDE, Rozmus J, Quinones R, Yu LC, Broglie L, Aquino V, Shereck E, Moore TB, Vander Lugt MT, Mousallem TI, Oved JH, Dorsey M, Abdel-Azim H, Martinez C, Bleesing JH, Prockop S, Kohn DB, Bednarski JJ, Leiding J, Marsh RA, Torgerson T, Notarangelo LD, Pai SY, Pulsipher MA, Puck JM, Dvorak CC, Haddad E, Buckley RH, Cowan MJ, Heimall J. Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study. J Allergy Clin Immunol. 2024 01; 153(1):287-296. View on PubMed
  20. Chandrasekaran P, Han Y, Zerbe CS, Heller T, DeRavin SS, Kreuzberg SA, Marciano BE, Siu Y, Jones DR, Abraham RS, Stephens MC, Tsou AM, Snapper S, Conlan S, Subramanian P, Quinones M, Grou C, Calderon V, Deming C, Leiding JW, Arnold DE, Logan BR, Griffith LM, Petrovic A, Mousallem TI, Kapoor N, Heimall JR, Barnum JL, Kapadia M, Wright N, Rayes A, Chandra S, Broglie LA, Chellapandian D, Deal CL, Grunebaum E, Lim SS, Mallhi K, Marsh RA, Murguia-Favela L, Parikh S, Touzot F, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Kang EM, Malech HL, Segre JA, Bryant CE, Holland SM, Falcone EL. Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease. J Allergy Clin Immunol. 2023 12; 152(6):1619-1633.e11. View on PubMed

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