Donald B Kohn Selected Research

Enzyme Replacement Therapy

10/2021	Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency.
10/2017	Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations.
5/2017	How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID).
11/2012	Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction.
9/2011	Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency.
6/2006	In vivo transduction by intravenous injection of a lentiviral vector expressing human ADA into neonatal ADA gene knockout mice: a novel form of enzyme replacement therapy for ADA deficiency.
11/2005	Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID).

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Donald B Kohn Research Topics

Disease

42	Severe Combined Immunodeficiency (Bare Lymphocyte Syndrome) 11/2023 - 10/2003
22	Neoplasms (Cancer) 05/2024 - 01/2002
20	Sickle Cell Anemia (Hemoglobin S Disease) 01/2024 - 07/2013
17	Severe combined immunodeficiency due to adenosine deaminase deficiency 11/2023 - 01/2003
16	Primary Immunodeficiency Diseases 10/2023 - 12/2008
11	Infections 12/2023 - 01/2002
8	Graft vs Host Disease (Graft-Versus-Host Disease) 04/2024 - 01/2003
7	Leukemia 10/2014 - 01/2002
6	Chronic Granulomatous Disease 01/2024 - 05/2010
6	Inborn Genetic Diseases (Disease, Hereditary) 01/2021 - 11/2015
6	Melanoma (Melanoma, Malignant) 01/2020 - 07/2008
5	X-Linked Combined Immunodeficiency Diseases (X Linked Severe Combined Immunodeficiency) 01/2021 - 05/2010
4	Lymphopenia (Lymphocytopenia) 11/2023 - 09/2011
3	Sarcoma (Soft Tissue Sarcoma) 10/2021 - 12/2002
3	Wiskott-Aldrich Syndrome 01/2021 - 01/2016
3	Prostatic Neoplasms (Prostate Cancer) 12/2013 - 07/2006
3	Precursor Cell Lymphoblastic Leukemia-Lymphoma (Acute Lymphoblastic Leukemia) 10/2005 - 01/2002
2	Exanthema (Rash) 01/2023 - 03/2009
2	Hematologic Neoplasms (Hematological Malignancy) 11/2022 - 06/2008
2	Anemia 06/2022 - 12/2018
2	COVID-19 12/2021 - 01/2021
2	Hemoglobinopathies 10/2020 - 01/2015
2	Pain (Aches) 12/2019 - 10/2016
2	Reticular dysgenesis 12/2017 - 04/2014
2	Vesicular Stomatitis 10/2014 - 10/2004
2	Philadelphia Chromosome 10/2005 - 01/2002
2	Mucopolysaccharidosis I (Hurler Syndrome) 05/2005 - 08/2003
1	Virus Diseases (Viral Diseases) 04/2024
1	Thrombotic Microangiopathies 04/2024
1	Inflammatory Bowel Diseases (Inflammatory Bowel Disease) 01/2024
1	Liver Diseases (Liver Disease) 12/2023
1	Eosinophilia 01/2023
1	Burkholderia Infections 01/2023
1	Lymphadenopathy 01/2023
1	Cytopenia 01/2022
1	Reinfection 12/2021
1	Pseudarthrosis (Pseudoarthrosis) 11/2021
1	Blast Injuries 11/2021
1	Open Fractures (Compound Fracture) 11/2021
1	Bruton type agammaglobulinemia 01/2021
1	Diarrhea 11/2020
1	Hypertension (High Blood Pressure) 11/2020
1	Pre-Eclampsia (Preeclampsia) 11/2020

Drug/Important Bio-Agent (IBA)

25	Adenosine DeaminaseIBA 11/2023 - 10/2003
14	GlobinsIBA 01/2023 - 07/2013
8	T-Cell Antigen Receptors (T-Cell Receptor)IBA 11/2016 - 12/2011
6	Busulfan (Busulfex)FDA Link 01/2024 - 02/2004
6	Hemoglobins (Hemoglobin)IBA 01/2023 - 01/2016
5	Chimeric Antigen ReceptorsIBA 05/2024 - 10/2013
5	VaccinesIBA 12/2021 - 07/2013
5	AntigensIBA 09/2014 - 07/2006
4	AntibodiesIBA 01/2022 - 01/2020
4	Proteins (Proteins, Gene)FDA Link 12/2021 - 02/2013
3	Oxidoreductases (Dehydrogenase)IBA 12/2023 - 01/2023
3	Codon (Codons)IBA 01/2023 - 03/2014
3	Complementary DNA (cDNA)IBA 10/2014 - 12/2002
2	Adrenal Cortex Hormones (Corticosteroids)IBA 04/2024 - 12/2023
2	SteroidsIBA 01/2024 - 12/2023
2	Adenine FDA LinkGeneric 11/2023 - 01/2023
2	HLA Antigens (Human Leukocyte Antigens)IBA 11/2021 - 05/2017
2	Immunoglobulins (Immunoglobulin)IBA 01/2021 - 08/2014
2	CD40 Ligand (CD40L)IBA 01/2018 - 01/2002
2	EnzymesIBA 10/2017 - 04/2014
2	Adenosine (Adenocard)FDA LinkGeneric 12/2015 - 11/2005
2	Alemtuzumab (Campath)FDA Link 11/2015 - 05/2007
2	Cyclophosphamide (Cytoxan)FDA LinkGeneric 11/2015 - 02/2004
2	Glycoproteins (Glycoprotein)IBA 10/2014 - 10/2004
2	Anti-Bacterial Agents (Antibiotics)IBA 08/2014 - 09/2011
2	Peptides (Polypeptides)IBA 05/2014 - 05/2013
2	HLA-A*02:01 antigenIBA 05/2013 - 12/2011
2	enhanced green fluorescent proteinIBA 02/2013 - 07/2003
2	Immunologic Factors (Immunomodulators)IBA 10/2005 - 01/2002
2	GlycosaminoglycansIBA 05/2005 - 08/2003
2	Iduronidase (alpha-L-Iduronidase)IBA 05/2005 - 08/2003
1	eculizumab FDA Link 04/2024
1	neutrophil cytosolic factor 1IBA 01/2024
1	NADP (NADPH)IBA 12/2023
1	Reactive Oxygen Species (Oxygen Radicals)IBA 12/2023
1	Immunoglobulin E (IgE)IBA 01/2023
1	deoxyhemoglobinIBA 01/2023
1	Amino Acids FDA Link 06/2022
1	Antiviral Agents (Antivirals)IBA 12/2021
1	Neutralizing AntibodiesIBA 12/2021
1	Phosphotransferases (Kinase)IBA 01/2021
1	Agammaglobulinaemia Tyrosine KinaseIBA 01/2021
1	Antihypertensive Agents (Antihypertensives)IBA 11/2020
1	ElectrolytesIBA 11/2020
1	SolutionsIBA 10/2020

Therapy/Procedure

22	Therapeutics 04/2024 - 02/2004
18	Hematopoietic Stem Cell Transplantation 10/2021 - 01/2002
14	Transplantation 01/2023 - 01/2002
12	Immunotherapy 05/2024 - 07/2006
11	Cell Transplantation 01/2024 - 12/2008
7	Drug Therapy (Chemotherapy) 11/2022 - 10/2005
7	Enzyme Replacement Therapy 10/2021 - 11/2005
3	Autologous Transplantation 10/2017 - 11/2012
2	Cell- and Tissue-Based Therapy (Cell Therapy) 11/2023 - 05/2014
2	Homologous Transplantation 05/2015 - 07/2013
1	Organ Transplantation 01/2023
1	Arthroplasty 11/2021