Page last updated: 2024-10-24

busulfan and Combined Immunodeficiency, X-Linked

busulfan has been researched along with Combined Immunodeficiency, X-Linked in 2 studies

Research Excerpts

ExcerptRelevanceReference
"Lentiviral vector gene therapy combined with low-exposure, targeted busulfan conditioning in infants with newly diagnosed SCID-X1 had low-grade acute toxic effects and resulted in multilineage engraftment of transduced cells, reconstitution of functional T cells and B cells, and normalization of NK-cell counts during a median follow-up of 16 months."2.90Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. ( Abdelsamed, H; Aldave Becerra, JC; Church, JA; Condori, J; Cowan, MJ; Cross, SJ; da Matta Ain, AC; De Ravin, SS; Dokmeci, E; Dowdy, J; Gottschalk, S; Janssen, W; Kang, G; Li, C; Lockey, T; Long-Boyle, JR; Love, JT; Ma, Z; Malech, HL; Mamcarz, E; Maron, G; Meagher, MM; Puck, JM; Ryu, BY; Sorrentino, BP; Tang, X; Triplett, B; van der Watt, H; Weiss, MJ; Youngblood, B; Zhou, S, 2019)

Research

Studies (2)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's0 (0.00)29.6817
2010's2 (100.00)24.3611
2020's0 (0.00)2.80

Authors

AuthorsStudies
Mamcarz, E1
Zhou, S1
Lockey, T1
Abdelsamed, H1
Cross, SJ1
Kang, G1
Ma, Z1
Condori, J1
Dowdy, J1
Triplett, B1
Li, C1
Maron, G1
Aldave Becerra, JC1
Church, JA1
Dokmeci, E1
Love, JT1
da Matta Ain, AC1
van der Watt, H1
Tang, X1
Janssen, W1
Ryu, BY1
De Ravin, SS1
Weiss, MJ1
Youngblood, B1
Long-Boyle, JR1
Gottschalk, S1
Meagher, MM1
Malech, HL1
Puck, JM1
Cowan, MJ1
Sorrentino, BP1

Clinical Trials (3)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
A Phase I/II Feasibility Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) Using a Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells[NCT03538899]Phase 1/Phase 225 participants (Anticipated)Interventional2018-05-31Recruiting
Gene Therapy for X Linked Severe Combined Immunodeficiency[NCT04286815]10 participants (Anticipated)Interventional2020-05-01Recruiting
A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells[NCT01512888]Phase 1/Phase 228 participants (Anticipated)Interventional2016-08-17Suspended (stopped due to voluntary hold)
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trials

1 trial available for busulfan and Combined Immunodeficiency, X-Linked

ArticleYear
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
    The New England journal of medicine, 2019, 04-18, Volume: 380, Issue:16

    Topics: Antigens, Differentiation, T-Lymphocyte; B-Lymphocytes; Busulfan; Genetic Therapy; Genetic Vectors;

2019
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
    The New England journal of medicine, 2019, 04-18, Volume: 380, Issue:16

    Topics: Antigens, Differentiation, T-Lymphocyte; B-Lymphocytes; Busulfan; Genetic Therapy; Genetic Vectors;

2019
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
    The New England journal of medicine, 2019, 04-18, Volume: 380, Issue:16

    Topics: Antigens, Differentiation, T-Lymphocyte; B-Lymphocytes; Busulfan; Genetic Therapy; Genetic Vectors;

2019
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
    The New England journal of medicine, 2019, 04-18, Volume: 380, Issue:16

    Topics: Antigens, Differentiation, T-Lymphocyte; B-Lymphocytes; Busulfan; Genetic Therapy; Genetic Vectors;

2019
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
    The New England journal of medicine, 2019, 04-18, Volume: 380, Issue:16

    Topics: Antigens, Differentiation, T-Lymphocyte; B-Lymphocytes; Busulfan; Genetic Therapy; Genetic Vectors;

2019
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
    The New England journal of medicine, 2019, 04-18, Volume: 380, Issue:16

    Topics: Antigens, Differentiation, T-Lymphocyte; B-Lymphocytes; Busulfan; Genetic Therapy; Genetic Vectors;

2019
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
    The New England journal of medicine, 2019, 04-18, Volume: 380, Issue:16

    Topics: Antigens, Differentiation, T-Lymphocyte; B-Lymphocytes; Busulfan; Genetic Therapy; Genetic Vectors;

2019
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
    The New England journal of medicine, 2019, 04-18, Volume: 380, Issue:16

    Topics: Antigens, Differentiation, T-Lymphocyte; B-Lymphocytes; Busulfan; Genetic Therapy; Genetic Vectors;

2019
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
    The New England journal of medicine, 2019, 04-18, Volume: 380, Issue:16

    Topics: Antigens, Differentiation, T-Lymphocyte; B-Lymphocytes; Busulfan; Genetic Therapy; Genetic Vectors;

2019

Other Studies

1 other study available for busulfan and Combined Immunodeficiency, X-Linked

ArticleYear
New Gene Therapy Potential Cure for "Bubble Boy Disease": An experimental gene therapy has allowed children with SCID-1X to develop fully functioning immune systems.
    American journal of medical genetics. Part A, 2019, Volume: 179, Issue:7

    Topics: Busulfan; Child; Child, Preschool; Female; Gene Expression; Genetic Therapy; Genetic Vectors; Humans

2019