Harry L. Malech

Also affiliated with Czech Academy of Sciences, Institute of Microbiology, National Institute of Allergy and Infectious Diseases, National Institute for the Psychotherapies

Immunology and MicrobiologyBiochemistry, Genetics and Molecular BiologyMedicine

Pilot corpus only

This score is computed over theSindex pilot corpus and does not cover the full scientific literature. Scores are relative to papers we have ingested — papers, authors, and institutions outside the pilot are not represented. Methodology.

Top 100%percentile

0Author DataRank

Indexed papers

0in pilot corpus

datarank_citation_only_1hop_v6· scope data_onlyMethodology

Papers

Driven by 53 papers. Top paper: “Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency”.

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

New England Journal of Medicine(2021)10.1056/nejmoa2027675

N/A

5.1DataRank · unranked

219 citations

Claire Booth, Kit L. Shaw, Jinhua Xu‐Bayford, Elizabeth Garabedian, Valentina Trevisan +48 more

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency

Blood(2021)10.1182/blood.2020010260

N/A

2.0DataRank · unranked

48 citations

Omar Habib, Kit L. Shaw, Elizabeth Garabedian, Denise A. Carbonaro-Sarracino, Dayna Terrazas +31 more

Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency

Nature Communications(2022)10.1038/s41467-022-31344-x

N/A

1.6DataRank · unranked

48 citations

Siyuan Liu, Colin L. Sweeney, Julie Brault, Narda Whiting‐Theobald, Michelle Ma +20 more

Clinical exome sequencing of 1000 families with complex immune phenotypes: Toward comprehensive genomic evaluations

Journal of Allergy and Clinical Immunology(2022)10.1016/j.jaci.2022.06.009

N/A

1.3DataRank · unranked

67 citations

Jia Yan, Rajarshi Ghosh, Andrew J. Oler, Luis M. Franco, Michael Setzer +95 more

A phase III randomized crossover trial of plerixafor versus G-CSF for treatment of WHIM syndrome

Journal of Clinical Investigation(2023)10.1172/jci164918

N/A

1.2DataRank · unranked

42 citations

Daniel Velez, Elena Cho, Edward W. Cowen, John J. DiGiovanna, Diana V. Pastrana +15 more

Evolution of Gene Therapy, Historical Perspective

Hematology/Oncology Clinics of North America(2022)10.1016/j.hoc.2022.05.001

N/A

0.950DataRank · unranked

44 citations

Elizabeth Garabedian, Matthew M. Hsieh, Harry L. Malech

Lentiviral gene therapy for X-linked chronic granulomatous disease

Nature Medicine(2020)10.1038/s41591-019-0735-5

N/A

0.832DataRank · unranked

256 citations

Claire Booth, Elizabeth M. Kang, Sung‐Yun Pai, Kit L. Shaw, Giorgia Santilli +33 more

Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Blood(2023)10.1182/blood.2022019586

N/A

0.816DataRank · unranked

26 citations

Danielle E. Arnold, Suhag Parikh, Brent R. Logan, Rebecca Marsh, Linda M. Griffith +64 more

Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease

Journal of Allergy and Clinical Immunology(2023)10.1016/j.jaci.2023.07.022

N/A

0.757DataRank · unranked

26 citations

Yu Han, Christa S. Zerbe, Theo Heller, Suk See De Ravin, Samantha A. Kreuzberg +52 more

The response to vedolizumab in chronic granulomatous disease-related inflammatory bowel disease

Gastroenterology report(2020)10.1093/gastro/goaa005

N/A

0.707DataRank · unranked

18 citations

Beatriz E. Marciano, Bryan F. Curtin, Anna Strongin, Suk See De Ravin, Athos Bousvaros +6 more

Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

Blood(2021)10.1182/blood.2020008503

N/A

0.650DataRank · unranked

75 citations

Julie Brault, Ronald J. Meis, Siyuan Liu, Linhong Li, Mara Pavel-Dinu +18 more

High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease

Science Translational Medicine(2024)10.1126/scitranslmed.adj6779

N/A

0.585DataRank · unranked

23 citations

Linyuan Ma, Siyuan Liu, Lauren R. Fox, Madelynn N. Whittaker, Ronald J. Meis +15 more

Gene Editing Rescues In vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System

Journal of Clinical Immunology(2021)10.1007/s10875-021-00989-6

N/A

0.568DataRank · unranked

43 citations

Mara Pavel-Dinu, Kerry Dobbs, Marita Bosticardo, Paul K. Reardon, Justin Lack +12 more

Failure to Prevent Severe Graft-Versus-Host Disease in Haploidentical Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide in Chronic Granulomatous Disease

Journal of Clinical Immunology(2020)10.1007/s10875-020-00772-z

N/A

0.489DataRank · unranked

25 citations

Dianne Hilligoss, Corin Kelly, Nana Kwatemaa, Narda Theobald, Christa S. Zerbe +4 more

Self-organized yolk sac-like organoids allow for scalable generation of multipotent hematopoietic progenitor cells from induced pluripotent stem cells

Cell Reports Methods(2023)10.1016/j.crmeth.2023.100460

N/A

0.449DataRank · unranked

19 citations

Stefan Siebert, Takuya Maeda, Ngoc‐Han Ha, Meghan L. Good, Yin Huang +21 more

Late-onset enteric virus infection associated with hepatitis (EVAH) in transplanted SCID patients

Journal of Allergy and Clinical Immunology(2023)10.1016/j.jaci.2022.12.822

N/A

0.425DataRank · unranked

16 citations

Jacques Fourgeaud, Julie Bruneau, Suk See De Ravin, Grace L. Smith, Mathieu Fusaro +53 more

Granulibacter bethesdensis, a Pathogen from Patients with Chronic Granulomatous Disease, Produces a Penta-Acylated Hypostimulatory Glycero-D-talo-oct-2-ulosonic Acid–Lipid A Glycolipid (Ko-Lipid A)

International Journal of Molecular Sciences(2021)10.3390/ijms22073303

N/A

0.345DataRank · unranked

9 citations

Kol A. Zarember, Christian Heiß, Joseph Shiloach, Lars J. Berg, John Audley +8 more

Outcomes in hematopoetic cell transplantation in the setting of mold infections in patients with chronic granulomatous disease

Bone Marrow Transplantation(2024)10.1038/s41409-024-02389-x

N/A

0.292DataRank · unranked

6 citations

Mark Parta, Jennifer Cuellar‐Rodríguez, Juan Gea‐Banacloche, Corin Kelly, Stefania Pittaluga +5 more

Progressive B Cell Loss in Revertant X-SCID

Journal of Clinical Immunology(2020)10.1007/s10875-020-00825-3

N/A

0.292DataRank · unranked

6 citations

Hye Sun Kuehn, Timothy J. Thauland, Christine Lee, Suk See De Ravin, Harry L. Malech +7 more

Small bowel disease prevalence on video capsule endoscopy in chronic granulomatous disease‐associated inflammatory bowel disease

Journal of Gastroenterology and Hepatology(2023)10.1111/jgh.16342

N/A

0.241DataRank · unranked

4 citations

Uni Wong, Mian Khalid, Hanna Blaney, Matthew G Menkart, Jenna L Oringher +8 more

Low Plasma Gelsolin Concentrations in Chronic Granulomatous Disease

Inflammation(2020)10.1007/s10753-020-01330-w

N/A

0.165DataRank · unranked

2 citations

Emily F. Gliniewicz, Kol A. Zarember, Hanna S. Hong, Gal Wald, Douglas B. Kuhns +9 more

Further Personalizing Medicine in Immune Disorders: Genomic Findings and Hematopoietic Cell Transplantation Survival

Transplantation(2025)10.1097/tp.0000000000005504

N/A

0.104DataRank · unranked

1 citations

Sarah A. Bannon, Jia Yan, Rajarshi Ghosh, Ekaterina Damskey, Breanna Beers +41 more

Umbilical Cord Blood Transplantation Provides an Alternative for Patients With Chronic Granulomatous Disease Lacking HLA-Matched Donors: A PIDTC Report

Transplantation and Cellular Therapy(2025)10.1016/j.jtct.2025.05.020

N/A

0.104DataRank · unranked

1 citations

Jennifer W. Leiding, Brent R. Logan, Rebecca Marsh, Linda M. Griffith, Eyal Grunebaum +36 more

Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human SCID-X1

Research Square(2021)10.21203/rs.3.rs-858393/v1

N/A

0DataRank · unranked

0 citations

Siyuan Liu, Colin L. Sweeney, Julie Brault, Narda Whiting‐Theobald, Michelle Ma +15 more

CRISPR-targetedMAGT1insertion restores XMEN patient hematopoietic stem cells and lymphocytes

Blood(2021)10.1182/blood.2021011192

N/A

0DataRank · unranked

38 citations

Taylor Liu, Ezekiel Bello, Siyuan Liu, Colin L. Sweeney, Ronald J. Meis +18 more

Hematologically important mutations: The autosomal forms of chronic granulomatous disease (third update)

Blood Cells Molecules and Diseases(2021)10.1016/j.bcmd.2021.102596

N/A

0DataRank · unranked

44 citations

Karin van Leeuwen, Amy P. Hsu, Debra Long Priel, Amber Begtrup, Rhonda Brandon +31 more

Correction: Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT

Journal of Clinical Immunology(2020)10.1007/s10875-020-00852-0

N/A

0DataRank · unranked

3 citations

Jennifer W. Leiding, Brent R. Logan, Linda M. Griffith, Danielle E. Arnold, Élie Haddad +50 more

Hematopoietic Stem Cell Transplantation in Late-onset X-linked Chronic Granulomatous Disease in a Female Carrier

Journal of Clinical Immunology(2022)10.1007/s10875-022-01310-9

N/A

0DataRank · unranked

6 citations

Elizabeth M. Kang, Francesco Salton, Barbara Ruaro, Chiara Torregiani, Paola Confalonieri +10 more

Febrile neutropenia management and outcomes in hematopoietic cell transplantation for chronic granulomatous disease

Transplant Infectious Disease(2022)10.1111/tid.13815

N/A

0DataRank · unranked

1 citations

Jennifer Cuellar‐Rodríguez, Juan Gea‐Banacloche, Jing Qin, Corin Kelly, Christa S. Zerbe +4 more

Genetically Corrected RAG2 -SCID Human Hematopoietic Stem Cells Restore V(D)J-Recombinase and Rescue Lymphoid Deficiency

bioRxiv (Cold Spring Harbor Laboratory)(2022)10.1101/2022.07.12.499831

N/A

0DataRank · unranked

3 citations

Cameron L. Gardner, Yusuke Nakauchi, Tomoki Kawai, Ottavia M. Delmonte, Boaz Palterer +16 more

Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study

Journal of Allergy and Clinical Immunology(2023)10.1016/j.jaci.2023.09.027

N/A

0DataRank · unranked

21 citations

Monica S. Thakar, Ami J. Shah, Brent R. Logan, Linda M. Griffith, Huaying Dong +53 more

Gastrointestinal and Hepatic Manifestations of Chronic Granulomatous Disease

The Journal of Allergy and Clinical Immunology In Practice(2023)10.1016/j.jaip.2022.12.039

N/A

0DataRank · unranked

20 citations

Brigit Sullivan, Christa S. Zerbe, Suk See De Ravin, Andrew M. Blakely, Martha Quezado +9 more

CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells

Frontiers in Immunology(2023)10.3389/fimmu.2022.1067417

N/A

0DataRank · unranked

26 citations

Taylor Liu, Siyuan Liu, Amanda Lawson, Uimook Choi, Nikita Kozhushko +16 more

Homozygous IL37 mutation associated with infantile inflammatory bowel disease

Proceedings of the National Academy of Sciences(2021)10.1073/pnas.2009217118

N/A

0DataRank · unranked

25 citations

Yu Zhang, Tingyan He, Colin L. Sweeney, Safa Barış, Elif Karakoç-Aydıner +10 more

Successful SCID gene therapy in infant with disseminated BCG

The Journal of Allergy and Clinical Immunology In Practice(2020)10.1016/j.jaip.2020.09.004

N/A

0DataRank · unranked

5 citations

Sue C. Kaste, Armita Bahrami, Michael D. Neel, Harry L. Malech, Jennifer M. Puck +4 more

MAGT1 messenger RNA-corrected autologous T and natural killer cells for potential cell therapy in X-linked immunodeficiency with magnesium defect, Epstein-Barr virus infection and neoplasia disease

Cytotherapy(2020)10.1016/j.jcyt.2020.08.013

N/A

0DataRank · unranked

15 citations

Ronald J. Meis, Linhong Li, Ezekiel Bello, Taylor Liu, Colin L. Sweeney +12 more

Allogeneic HSCT for Symptomatic Female X-linked Chronic Granulomatous Disease Carriers

Journal of Clinical Immunology(2023)10.1007/s10875-023-01570-z

N/A

0DataRank · unranked

13 citations

Tuulia Torppa, Eleri Williams, Michael H. Albert, Fabian Hauck, Elena Soncini +7 more

Gene therapy for inborn errors of immunity: Base editing comes into play

Cell(2023)10.1016/j.cell.2023.03.001

N/A

0DataRank · unranked

13 citations

Luigi D. Notarangelo, Harry L. Malech

Genetically corrected RAG2 -SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency

Blood Advances(2023)10.1182/bloodadvances.2023011766

N/A

0DataRank · unranked

13 citations

Cameron L. Gardner, Yusuke Nakauchi, Tomoki Kawai, Ottavia M. Delmonte, Boaz Palterer +18 more

CRISPR/Cas9-mediated Cxcr4 Disease Allele Inactivation for Gene Therapy in a Mouse Model of WHIM Syndrome

Blood(2023)10.1182/blood.2022019142

N/A

0DataRank · unranked

2 citations

Albert Owusu-Ansah, Alexander Yang, Erin Yim, David H. McDermott, P Jacobs +6 more

Severe combined immunodeficiency: improved survival leading to detection of underlying liver disease

BMC Gastroenterology(2023)10.1186/s12876-023-02782-8

N/A

0DataRank · unranked

0 citations

Nehna Abdul Majeed, Elizabeth Garabedian, Jamie Marko, David E. Kleiner, Robert Sokolic +5 more

Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency

Science Translational Medicine(2024)10.1126/scitranslmed.adh8162

N/A

0DataRank · unranked

23 citations

Chiara Brandas, Samuele Ferrari, Simona Porcellini, Nicolò Sacchetti, Daniele Canarutto +29 more

Artificial thymic organoids represent a reliable tool to study T-cell differentiation in patients with severe T-cell lymphopenia

Blood Advances(2020)10.1182/bloodadvances.2020001730

N/A

0DataRank · unranked

100 citations

Francesca Pala, Enrica Calzoni, Ottavia M. Delmonte, Kerry Dobbs, Cameron L. Gardner +20 more

High symptom burden in female X-linked chronic granulomatous disease carriers

Clinical Immunology(2024)10.1016/j.clim.2024.110364

N/A

0DataRank · unranked

11 citations

Athanasios Tsalatsanis, Jessica Trotter, Danielle E. Arnold, Jacqueline D. Squire, Sharon A. Kidd +24 more

Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A Primary Immune Deficiency Treatment Consortium study

Journal of Allergy and Clinical Immunology(2024)10.1016/j.jaci.2024.01.013

N/A

0DataRank · unranked

10 citations

Danielle E. Arnold, Brent R. Logan, Suhag Parikh, Rebecca Marsh, Linda M. Griffith +36 more

Advancing gene-editing platforms to improve the viability of rare-disease therapeutics: key insights from a 2024 Scientific Exchange hosted by ARM, ISCT, and Danaher

Cytotherapy(2025)10.1016/j.jcyt.2025.06.010

N/A

0DataRank · unranked

8 citations

Sadik H. Kassim, Kiran Musunuru, David R. Liu, Ann Y. Lee, Luis Barrera +14 more

Reliable genetic diagnosis of NCF1 (p47phox)-deficient chronic granulomatous disease using high-throughput sequencing

Frontiers in Immunology(2025)10.3389/fimmu.2025.1640496

N/A

0DataRank · unranked

1 citations

Eric Karlins, Justin Lack, Thomas Pepper, Karen Lau, Kimberly R. Marshall‐Batty +9 more

NADPH oxidase correction by mRNA transfection of apheresis granulocytes in chronic granulomatous disease

Blood Advances(2020)10.1182/bloodadvances.2020003224

N/A

0DataRank · unranked

4 citations

Julie Brault, Ronald J. Meis, Linhong Li, Narda Theobald, Aylin Bonifacino +21 more

Homozygous variant p. Arg90His in NCF1 is associated with early-onset Interferonopathy: a case report

Pediatric Rheumatology(2021)10.1186/s12969-021-00536-y

N/A

0DataRank · unranked

12 citations

Liane Heale, Dilan Dissanayake, Wanxia Li Tsai, Massimo Gadina, Thomas L. Leto +6 more

Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47 phox -Deficient Chronic Granulomatous Disease

Human Gene Therapy(2021)10.1089/hum.2020.276

N/A

0DataRank · unranked

10 citations

Winston Vetharoy, Uimook Choi, Christine Rivat, Narda Theobald, Giuseppa Piras +13 more

Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models

Cell Reports Medicine(2021)10.1016/j.xcrm.2021.100247

N/A

0DataRank · unranked

27 citations

Linhong Li, Tina Nassehi, Claire Drysdale, Morgan Yapundich, Jackson Gamer +13 more

Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair

Gene Therapy(2021)10.1038/s41434-021-00251-z

N/A

0DataRank · unranked

52 citations

Mara Pavel-Dinu, Uimook Choi, Julie Brault, Taylor Liu, Sherry Koontz +11 more

Self-organized yolk sac-like organoids allow for scalable generation of multipotent hematopoietic progenitor cells from human induced pluripotent stem cells

bioRxiv (Cold Spring Harbor Laboratory)(2021)10.1101/2021.04.25.441298

N/A

0DataRank · unranked

2 citations

Stefan Siebert, Takuya Maeda, Ngoc‐Han Ha, Meghan L. Good, Yin Huang +21 more