ArticleThe New England journal of medicine2025
Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A.
Registry-linked trialAbstract readClinical Trial, Phase I
In one paragraph
Article in The New England journal of medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05265767 (Gene Therapy for Hemophilia A With a High Expression Factor VIII Transgene in Autologous Hematopoietic Stem Cells), which is not on this map. Cited by 37 papers.
0numbers the graph read from it
0cells of the map it votes in
37citing papers in PubMed
–field-weighted citation impact
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Gene Therapy for Hemophilia A With a High Expression Factor VIII Transgene in Autologous Hematopoietic Stem Cells
Who cites it
37 citing papers in PubMed.
- Antimetabolites synergize with non-genotoxic antibody drug conjugate conditioning in hematopoietic stem cell lentiviral gene therapy.Molecular therapy. Advances · 2026Article
- Continuum of Care for Hemophilia: The Story of India.Indian journal of pediatrics · 2026Review
- Democratizing the Cure: Indigenous Innovations and the Future of Pediatric Cell and Gene Therapy in India.Indian pediatrics · 2026Review
- Red lines and green lights: Gene therapy for inherited erythroid disorders beyond the haemoglobinopathies.British journal of haematology · 2026Review
- Gene therapy for liver diseases: methods, challenges and opportunities.Journal of nanobiotechnology · 2026Review
- "Primed for Repair: Harnessing Hypoxia, Mechanobiology, and Gene Editing to Enhance MSC Potency and Clinical Translation".Stem cell reviews and reports · 2026Review
- Evolving landscape of haemophilia care in India: Balancing innovation, access, and sustainability.The Indian journal of medical research · 2026Article
- Transforming Hemophilia Management: Lessons from Gene Therapy Clinical Trials.Molecular biotechnology · 2026Review
- Humanized mice enableMolecular therapy. Advances · 2026Article
- Hemophilia and hereditary angioedema: parallel therapeutic advances in genetic diseases of serine protease pathways.Research and practice in thrombosis and haemostasis · 2026Review
- Treatment of canine hemophilia A via intraosseous delivery of a platelet-specific factor VIII-lentiviral vector.Blood vessels, thrombosis & hemostasis · 2026Article
- Hematopoietic (stem) cells-The elixir of life?FEBS letters · 2026Review
- Advancements in CRISPR-basedFrontiers in genome editing · 2026Review
- Nine areas with outstanding challenges for hemophilia B research.Therapeutic advances in hematology · 2026Review
- Current Status of Clinical Gene Therapy for Hemophilia and Globin Disorders.Journal of blood medicine · 2026Review
- Balancing Promise and Peril: Hemophilia Gene Therapy Insights.IUBMB life · 2026Review
- Deconstructing gene therapy in hemophilia for the clinician.Hematology. American Society of Hematology. Education Program · 2025Review
- Robot-assisted total hip arthroplasty following gene therapy for hemophilia a arthropathy: a case report.Arthroplasty (London, England) · 2025Article
- How Close Are We to Achieving Durable and Efficacious Gene Therapy for Hemophilia A and B?Genes · 2025Review
- Therapeutic innovations in hemophilia: the essential role of a positive reinvestment cycle.Blood advances · 2025Review
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Authors and funding
23 authors.
Alok SrivastavaFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Aby AbrahamFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Fouzia AboobackerFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Gurbind SinghFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Tulasi GeevarFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Uday KulkarniFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).ORCID 0000-0003-2230-2424
Sushil SelvarajanFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Anu KorulaFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Rutvi Gautam DaveFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Mohana ShankarFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Abraham S SinghFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Anbu JebaFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Navien KumaarFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Christopher BenjaminFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Kavitha M LakshmiFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Vivi Miriam SrivastavaFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Ramachandran V ShajiFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Sukesh C NairFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Harrison C BrownFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Gabriela DenningFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Pete LollarFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Christopher B DoeringFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Trent SpencerFrom the Department of Hematology, Christian Medical College Vellore, Ranipet Campus, Vellore, India (A.S., A.A., F.A., U.K., S.S., A.K., A.S.S., A.J., N.K., C.B., K.M.L., R.V.S.); Center for Stem Cell Research Unit of inStem, Bengaluru, Christian Medical College Vellore, Vellore, India (A.S., G.S., M.S., R.V.S.); the Department of Immunohematology and Transfusion Medicine, Christian Medical College Vellore, Town Campus, Vellore, India (T.G., R.G.D., S.C.N.); the Department of Cytogenetics, Christian Medical College Vellore, Vellore, India (V.M.S.); Expression Therapeutics, Tucker, GA (H.C.B., G.D., P.L., C.B.D., T.S.); and Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta (P.L., C.B.D., T.S.).
Funding
Translational Research SkillsU54HL112309 · NHLBI · EMORY UNIVERSITY · PI LOLLAR, JOHN S. · 2012 to 2016
$12.1MSTRUCTURE/FUNCTION OF THE FACTOR VIII VWF COMPLEXR01HL040921 · NHLBI · UNIVERSITY OF VERMONT &ST AGRIC COLLEGE · PI LOLLAR, JOHN S. · 1988 to 2010
$2.5MBioengineered factor VIII gene therapy for hemophilia AR44HL114241 · NHLBI · EXPRESSION THERAPEUTICS · PI DENNING, GABRIELA, DOERING, CHRISTOPHER BRADLEY · 2014 to 2015
$1.5MAncestral Sequence Reconstruction Engineering of Coagulation FactorsR01HL137128 · NHLBI · EMORY UNIVERSITY · PI DOERING, CHRISTOPHER BRADLEY, SPENCER, H TRENT · 2017 to 2019
$1.1MBioengineering Factor VIII through Ancestral ReconstructionR56HL131059 · NHLBI · EMORY UNIVERSITY · PI DOERING, CHRISTOPHER BRADLEY, SPENCER, H TRENT · 2016 to 2016
$385kBioengineered factor VIII gene therapy for hemophilia AR43HL114241 · NHLBI · EXPRESSION THERAPEUTICS · PI DENNING, GABRIELA, DOERING, CHRISTOPHER BRADLEY · 2012 to 2012
$378kDepartment of Biotechnology, Ministry of Science and Technology, India BT/PR17316/MED/31/326/2015Hemophilia of Georgia 107993NHLBI NIH HHS HL112309, HL131059, HL137128, HL 114241NHLBI NIH HHS R01 HL040921NHLBI NIH HHS R01 HL137128NHLBI NIH HHS R43 HL114241NHLBI NIH HHS R44 HL114241NHLBI NIH HHS R56 HL131059NHLBI NIH HHS U54 HL112309
Abstract
backgroundSevere hemophilia A is managed with factor VIII replacement or hemostatic products that stop or prevent bleeding. Data on gene therapy with hematopoietic stem-cell (HSC)-based expression of factor VIII for the treatment of severe hemophilia A are lacking.
methodsWe conducted a single-center study involving five participants 22 to 41 years of age with severe hemophilia A without factor VIII inhibitors. Autologous HSCs were transduced with CD68-ET3-LV - a lentiviral vector including a new
resultsParticipants received CD68-ET3-LV-transduced autologous CD34+ HSCs at doses of 5.0×10
conclusionsGene therapy for hemophilia A with the use of lentiviral vector-transduced autologous HSCs resulted in stable factor VIII expression, with factor VIII activity correlating to vector copy number in the peripheral blood. (Funded by the Ministry of Science and Technology, Government of India, and others; ClinicalTrials.gov number, NCT05265767; Clinical Trials Registry-India number, CTRI/2022/03/041304.).
Indexed as
Factor VIIIGenetic TherapyHematopoietic Stem Cell TransplantationHemophilia AHemorrhageAdultAntigens, CD34Follow-Up StudiesGenetic VectorsHematopoietic Stem CellsHumansLentivirusMaleTransduction, GeneticTransgenesTransplantation, AutologousAntigens, CD34Factor VIII
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Registered trials
NCT05265767ClinicalTrials.gov
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