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Gene Therapy for Transfusion Dependent Beta-thalassemia (TIGET-BTHAL)

Primary Purpose

Beta-Thalassemia

Status
Unknown status
Phase
Phase 1
Locations
Italy
Study Type
Interventional
Intervention
Autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene
Sponsored by
IRCCS San Raffaele
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Beta-Thalassemia focused on measuring Beta thalassemia, Gene therapy, Lentiviral vector

Eligibility Criteria

3 Years - 64 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Written informed consent
  • Transfusion-dependent beta-thalassemia (any genotype). Transfusion dependence is defined as receiving ≥ 8 transfusions of blood per year over a minimum of 2 years.
  • Karnofsky Index or Lansky > 80%
  • Age ≥ 3 years and < 65 years

  • Adequate cardiac, renal, hepatic and pulmonary functions as evidenced by:

    • Left ventricular ejection fraction (LVEF) greater than 45% by echo and normal ECG or presence of abnormalities not significant for cardiac disease. Absence of severe pulmonary hypertension
    • Diffusing capacity of the lung for carbon monoxide (DLCO) > 50% and forced expiratory volume in 1 sec (FEV1) and forced expiratory vital capacity (FVC) > 60% predicted (if non cooperative: pulse oximetry > 95 % in room air)
    • Serum creatinine < 1.5 upper limit of normal
    • Absent-mild-moderate liver iron overload on T2*MRI (less than 12 months before enrolment)
    • Absent-mild-moderate cardiac iron overload T2*MRI (less than 12 months before enrolment)
    • Absence of severe liver fibrosis or cirrhosis on fibroscan or liver biopsy (less than 12 months before enrolment)
  • Low risk thrombophilic screen and negative history of significant previous thrombotic events
  • For all patients in reproductive age, agreement to use highly effective and adequate method of contraception while receiving treatment phase and for at least 12 months following drugs administration (including both females of child bearing potential and males with partners of child bearing potential)
  • Good adherence to transfusion and chelation programme as indirect evidence of good adherence to treatment and follow-up evaluations for current trial
  • Availability of an adequate and well documented transfusion history (at least previous 6 months) or availability to follow a regular transfusion regimen according to guidelines and provide a detailed transfusion record of the 6 months prior to intervention phase

Exclusion Criteria:

  • Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents)
  • Severe, active viral, bacterial, or fungal infection at eligibility evaluation
  • Malignant neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or exceptional family history of familial cancer syndromes
  • Myelodysplasia, cytogenetic alterations associated with neoplasia, or other serious haematological disorder than thalassemia
  • History of uncontrolled seizures
  • Other clinical conditions judged non compatible with the procedure and/or the treatment
  • Positivity for HIV (serology or RNA), and/or HbsAg and/or HBV DNA and/or HCV RNA (or negative HCV RNA but on antiviral treatment) and/or Treponema Pallidum or Mycoplasma active infection
  • Active alcohol or substance abuse within 6 months of the study
  • Pregnancy or lactation
  • Previous allogeneic bone marrow transplantation or gene therapy
  • For paediatric patients only: availability of an HLA-matched donor (sibling or of a suitable 10/10 matched unrelated donor).

Sites / Locations

  • Ospedale San Raffaele

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

Adults

Elderly children

Younger children

Arm Description

≥18 years (3 subjects) The ATIMP consists of autologous CD34+ cell enriched fraction containing hematopoietic stem cells (HSC) transduced with the GLOBE lentiviral vector encoding for the beta-globin gene re-suspended in their final formulation medium. Dosage indications The target dose in the transduced product is 5x10(6) cells/Kg CD34+cells, with a minimum dose of 2x10(6)/Kg and a maximum dose of 20x10(6)/Kg, depending on the yield of cells. The product will be injected intraosseously.

8-17 years (3 subjects) The ATIMP consists of autologous CD34+ cell enriched fraction containing hematopoietic stem cells (HSC) transduced with the GLOBE lentiviral vector encoding for the beta-globin gene re-suspended in their final formulation medium. Dosage indications The target dose in the transduced product is 5x10(6) cells/Kg CD34+cells, with a minimum dose of 2x10(6)/Kg and a maximum dose of 20x10(6)/Kg, depending on the yield of cells. The product will be injected intraosseously.

3-7 years (4 subjects) The ATIMP consists of autologous CD34+ cell enriched fraction containing hematopoietic stem cells (HSC) transduced with the GLOBE lentiviral vector encoding for the beta-globin gene re-suspended in their final formulation medium. Dosage indications The target dose in the transduced product is 5x10(6) cells/Kg CD34+cells, with a minimum dose of 2x10(6)/Kg and a maximum dose of 20x10(6)/Kg, depending on the yield of cells. The product will be injected intraosseously.

Outcomes

Primary Outcome Measures

Overall survival
Number of patients alive all over the trial
Achievement of hematological engraftment
Haematological engraftment is defined as first day of neutrophil count >500/mm3 and platelets >20,000/mm3 on 3 consecutive blood counts.
Safety of the administration of autologous haematopoietic stem cells transduced with LV-GLOBE
short-term tolerability: the percentage of patients not experiencing short-term (0-24 hours from injection) adverse events (of any grade) and systemic reactions. absence of Replication Competent Lentivirus (RCL): the percentage of subjects without RCL in the 24 months from injection. absence of abnormal clonal proliferation: the percentage of subjects without abnormal clonal proliferation in the 24 months from injection.
Short-term safety and tolerability of the different conditioning regimens
The percentage of patients with the following clinical events from day -5 to +100 days from injection: NCI (National Cancer Institute Common Terminology Criteria grading) ≥2 and metabolic/laboratory NCI ≥3.
Overall safety and tolerability measured by AE recording
The number of AEs (adverse events) and SAEs (serious adverse events) and the percentage of subjects experiencing AEs and SAEs in the 24 months post injection will be summarized by severity and within body system involved.
Polyclonal engraftment
The percentage of subjects with polyclonality of haematopoiesis will be estimated at 6, 12, 18 and 24 months from injection. Polyclonality of haematopoiesis will be defined as > 1000 unique integration sites retrieved from peripheral blood and/or bone marrow cells.
Reduction in transfusion frequency up to transfusion independence
Transfusions will be recorded as mLs of blood/kg/months

Secondary Outcome Measures

Transfusion independence
Transfusion independence is defined as ≤ 1 transfusion in the previous 6 months
Adequate haemoglobin level
Haemoglobin level will be assessed by full blood counts in patients achieving transfusion independence. Adequate haemoglobin is defined as haemoglobin >9 g/dl in adults and >10 g/dl in children.
Adequate engraftment of genetically corrected cells
Engraftment will be assessed by vector-specific quantitative Polymerase Chain reaction (PCR) on bone marrow. Adequate engraftment is defined as ≥ 0.15 VCN/genome. (VCN = Vector Copy Number)
Transgene expression
Transgene expression will be evaluated by globin chains and/or hemoglobin synthesis on peripheral blood and/or bone marrow samples by HPLC and/or electrophoresis analysis
Improvement of health-related quality of life
Health-related quality of life will be assessed by the use of standardized questionnaires

Full Information

First Posted
May 15, 2015
Last Updated
June 26, 2019
Sponsor
IRCCS San Raffaele
Collaborators
Fondazione Telethon, Orchard Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT02453477
Brief Title
Gene Therapy for Transfusion Dependent Beta-thalassemia
Acronym
TIGET-BTHAL
Official Title
A Phase I/II Study Evaluating Safety and Efficacy of Autologous Hematopoietic Stem Cells Genetically Modified With GLOBE Lentiviral Vector Encoding for the Human Beta-globin Gene for the Treatment of Patients Affected by Transfusion Dependent Beta-thalassemia
Study Type
Interventional

2. Study Status

Record Verification Date
June 2019
Overall Recruitment Status
Unknown status
Study Start Date
May 2015 (undefined)
Primary Completion Date
August 2019 (Anticipated)
Study Completion Date
August 2019 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
IRCCS San Raffaele
Collaborators
Fondazione Telethon, Orchard Therapeutics

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by transfusion dependent beta-thalassemia
Detailed Description
Both adults and pediatric patients will be treated with genetically modified autologous hematopoietic stem cells collected from mobilized peripheral blood (or bone marrow for patients < 8 years in case mobilization will not be feasible) and transduced with GLOBE lentiviral vector encoding for the human beta-globin gene. This study will enroll 10 patients allocated in 3 groups, according to age and conditioning regimen: 3 adults (≥18 years) conditioned with treosulfan and thiotepa 3 elderly children (8-17 years) conditioned with treosulfan and thiotepa 4 younger children (3-7 years) conditioned with treosulfan and thiotepa Enrolment will start in adult patients. Pediatric patients will be included once evidence of preliminary safety and biological efficacy is shown in at least 2 adults. Patients are included regardless of the beta globin gene mutation, provided an adequate cardiac, renal, hepatic and pulmonary function is demonstrated. Patients with severe iron overload are excluded as well as patients with active viral infections. Pediatric patients can be enrolled only in absence of a human leukocyte antigen (HLA)-identical sibling or a suitable 10/10 matched unrelated donor. The treated patients will be followed for 2 years. After completion of the 2 years follow up, patients will be enrolled in a long term follow up study and followed up for at least other additional 6 years.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Beta-Thalassemia
Keywords
Beta thalassemia, Gene therapy, Lentiviral vector

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Adults
Arm Type
Experimental
Arm Description
≥18 years (3 subjects) The ATIMP consists of autologous CD34+ cell enriched fraction containing hematopoietic stem cells (HSC) transduced with the GLOBE lentiviral vector encoding for the beta-globin gene re-suspended in their final formulation medium. Dosage indications The target dose in the transduced product is 5x10(6) cells/Kg CD34+cells, with a minimum dose of 2x10(6)/Kg and a maximum dose of 20x10(6)/Kg, depending on the yield of cells. The product will be injected intraosseously.
Arm Title
Elderly children
Arm Type
Experimental
Arm Description
8-17 years (3 subjects) The ATIMP consists of autologous CD34+ cell enriched fraction containing hematopoietic stem cells (HSC) transduced with the GLOBE lentiviral vector encoding for the beta-globin gene re-suspended in their final formulation medium. Dosage indications The target dose in the transduced product is 5x10(6) cells/Kg CD34+cells, with a minimum dose of 2x10(6)/Kg and a maximum dose of 20x10(6)/Kg, depending on the yield of cells. The product will be injected intraosseously.
Arm Title
Younger children
Arm Type
Experimental
Arm Description
3-7 years (4 subjects) The ATIMP consists of autologous CD34+ cell enriched fraction containing hematopoietic stem cells (HSC) transduced with the GLOBE lentiviral vector encoding for the beta-globin gene re-suspended in their final formulation medium. Dosage indications The target dose in the transduced product is 5x10(6) cells/Kg CD34+cells, with a minimum dose of 2x10(6)/Kg and a maximum dose of 20x10(6)/Kg, depending on the yield of cells. The product will be injected intraosseously.
Intervention Type
Genetic
Intervention Name(s)
Autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene
Intervention Description
Autologous CD34+ cell enriched fraction containing hematopoietic stem cells (HSC) transduced with the GLOBE lentiviral vector encoding for the human beta-globin gene resuspended in their final formulation medium. The target dose in the transduced product is 5x10^6 cells/Kg CD34+ cells, with a minimum dose of 2 x 10^6/Kg and a maximum dose of 20 x 10^6/Kg, depending on the yield of cells. The product will be injected intraosseously.
Primary Outcome Measure Information:
Title
Overall survival
Description
Number of patients alive all over the trial
Time Frame
2 years
Title
Achievement of hematological engraftment
Description
Haematological engraftment is defined as first day of neutrophil count >500/mm3 and platelets >20,000/mm3 on 3 consecutive blood counts.
Time Frame
within day +60 after gene therapy
Title
Safety of the administration of autologous haematopoietic stem cells transduced with LV-GLOBE
Description
short-term tolerability: the percentage of patients not experiencing short-term (0-24 hours from injection) adverse events (of any grade) and systemic reactions. absence of Replication Competent Lentivirus (RCL): the percentage of subjects without RCL in the 24 months from injection. absence of abnormal clonal proliferation: the percentage of subjects without abnormal clonal proliferation in the 24 months from injection.
Time Frame
0-24 months after gene therapy
Title
Short-term safety and tolerability of the different conditioning regimens
Description
The percentage of patients with the following clinical events from day -5 to +100 days from injection: NCI (National Cancer Institute Common Terminology Criteria grading) ≥2 and metabolic/laboratory NCI ≥3.
Time Frame
from day -5 (first day of conditioning treatment) to day 100 after gene therapy
Title
Overall safety and tolerability measured by AE recording
Description
The number of AEs (adverse events) and SAEs (serious adverse events) and the percentage of subjects experiencing AEs and SAEs in the 24 months post injection will be summarized by severity and within body system involved.
Time Frame
0-24 months after gene therapy
Title
Polyclonal engraftment
Description
The percentage of subjects with polyclonality of haematopoiesis will be estimated at 6, 12, 18 and 24 months from injection. Polyclonality of haematopoiesis will be defined as > 1000 unique integration sites retrieved from peripheral blood and/or bone marrow cells.
Time Frame
From 6 months to 2 years after gene therapy
Title
Reduction in transfusion frequency up to transfusion independence
Description
Transfusions will be recorded as mLs of blood/kg/months
Time Frame
from -7 months before gene therapy to 2 years after gene therapy
Secondary Outcome Measure Information:
Title
Transfusion independence
Description
Transfusion independence is defined as ≤ 1 transfusion in the previous 6 months
Time Frame
9 months, 1, 1.5 and 2 years after gene therapy
Title
Adequate haemoglobin level
Description
Haemoglobin level will be assessed by full blood counts in patients achieving transfusion independence. Adequate haemoglobin is defined as haemoglobin >9 g/dl in adults and >10 g/dl in children.
Time Frame
0-24 months after gene therapy
Title
Adequate engraftment of genetically corrected cells
Description
Engraftment will be assessed by vector-specific quantitative Polymerase Chain reaction (PCR) on bone marrow. Adequate engraftment is defined as ≥ 0.15 VCN/genome. (VCN = Vector Copy Number)
Time Frame
6, 12, and 24 months after gene therapy
Title
Transgene expression
Description
Transgene expression will be evaluated by globin chains and/or hemoglobin synthesis on peripheral blood and/or bone marrow samples by HPLC and/or electrophoresis analysis
Time Frame
6, 12, and 24 months after gene therapy
Title
Improvement of health-related quality of life
Description
Health-related quality of life will be assessed by the use of standardized questionnaires
Time Frame
12 and 24 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
64 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Written informed consent Transfusion-dependent beta-thalassemia (any genotype). Transfusion dependence is defined as receiving ≥ 8 transfusions of blood per year over a minimum of 2 years. Karnofsky Index or Lansky > 80% Age ≥ 3 years and < 65 years Adequate cardiac, renal, hepatic and pulmonary functions as evidenced by: Left ventricular ejection fraction (LVEF) greater than 45% by echo and normal ECG or presence of abnormalities not significant for cardiac disease. Absence of severe pulmonary hypertension Diffusing capacity of the lung for carbon monoxide (DLCO) > 50% and forced expiratory volume in 1 sec (FEV1) and forced expiratory vital capacity (FVC) > 60% predicted (if non cooperative: pulse oximetry > 95 % in room air) Serum creatinine < 1.5 upper limit of normal Absent-mild-moderate liver iron overload on T2*MRI (less than 12 months before enrolment) Absent-mild-moderate cardiac iron overload T2*MRI (less than 12 months before enrolment) Absence of severe liver fibrosis or cirrhosis on fibroscan or liver biopsy (less than 12 months before enrolment) Low risk thrombophilic screen and negative history of significant previous thrombotic events For all patients in reproductive age, agreement to use highly effective and adequate method of contraception while receiving treatment phase and for at least 12 months following drugs administration (including both females of child bearing potential and males with partners of child bearing potential) Good adherence to transfusion and chelation programme as indirect evidence of good adherence to treatment and follow-up evaluations for current trial Availability of an adequate and well documented transfusion history (at least previous 6 months) or availability to follow a regular transfusion regimen according to guidelines and provide a detailed transfusion record of the 6 months prior to intervention phase Exclusion Criteria: Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents) Severe, active viral, bacterial, or fungal infection at eligibility evaluation Malignant neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or exceptional family history of familial cancer syndromes Myelodysplasia, cytogenetic alterations associated with neoplasia, or other serious haematological disorder than thalassemia History of uncontrolled seizures Other clinical conditions judged non compatible with the procedure and/or the treatment Positivity for HIV (serology or RNA), and/or HbsAg and/or HBV DNA and/or HCV RNA (or negative HCV RNA but on antiviral treatment) and/or Treponema Pallidum or Mycoplasma active infection Active alcohol or substance abuse within 6 months of the study Pregnancy or lactation Previous allogeneic bone marrow transplantation or gene therapy For paediatric patients only: availability of an HLA-matched donor (sibling or of a suitable 10/10 matched unrelated donor).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Alessandro Aiuti, MD, PhD
Organizational Affiliation
Ospedale San Raffaele
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Fabio Ciceri, MD
Organizational Affiliation
Ospedale San Raffaele
Official's Role
Study Chair
First Name & Middle Initial & Last Name & Degree
Sarah Marktel, MD
Organizational Affiliation
Ospedale San Raffaele
Official's Role
Study Chair
First Name & Middle Initial & Last Name & Degree
Maria Domenica Cappellini, MD
Organizational Affiliation
IRCCS Policlinico Foundation
Official's Role
Study Chair
First Name & Middle Initial & Last Name & Degree
Giuliana Ferrari, PhD
Organizational Affiliation
Telethon Institute of Gene Therapy, Ospedale San Raffaele
Official's Role
Study Director
Facility Information:
Facility Name
Ospedale San Raffaele
City
Milano
ZIP/Postal Code
20132
Country
Italy

12. IPD Sharing Statement

Citations:
PubMed Identifier
23845947
Citation
Aiuti A, Biasco L, Scaramuzza S, Ferrua F, Cicalese MP, Baricordi C, Dionisio F, Calabria A, Giannelli S, Castiello MC, Bosticardo M, Evangelio C, Assanelli A, Casiraghi M, Di Nunzio S, Callegaro L, Benati C, Rizzardi P, Pellin D, Di Serio C, Schmidt M, Von Kalle C, Gardner J, Mehta N, Neduva V, Dow DJ, Galy A, Miniero R, Finocchi A, Metin A, Banerjee PP, Orange JS, Galimberti S, Valsecchi MG, Biffi A, Montini E, Villa A, Ciceri F, Roncarolo MG, Naldini L. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science. 2013 Aug 23;341(6148):1233151. doi: 10.1126/science.1233151. Epub 2013 Jul 11.
Results Reference
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PubMed Identifier
17671653
Citation
Aiuti A, Cassani B, Andolfi G, Mirolo M, Biasco L, Recchia A, Urbinati F, Valacca C, Scaramuzza S, Aker M, Slavin S, Cazzola M, Sartori D, Ambrosi A, Di Serio C, Roncarolo MG, Mavilio F, Bordignon C. Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. J Clin Invest. 2007 Aug;117(8):2233-40. doi: 10.1172/JCI31666.
Results Reference
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PubMed Identifier
19179314
Citation
Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, Scaramuzza S, Andolfi G, Mirolo M, Brigida I, Tabucchi A, Carlucci F, Eibl M, Aker M, Slavin S, Al-Mousa H, Al Ghonaium A, Ferster A, Duppenthaler A, Notarangelo L, Wintergerst U, Buckley RH, Bregni M, Marktel S, Valsecchi MG, Rossi P, Ciceri F, Miniero R, Bordignon C, Roncarolo MG. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med. 2009 Jan 29;360(5):447-58. doi: 10.1056/NEJMoa0805817.
Results Reference
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PubMed Identifier
23845948
Citation
Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T, Baldoli C, Martino S, Calabria A, Canale S, Benedicenti F, Vallanti G, Biasco L, Leo S, Kabbara N, Zanetti G, Rizzo WB, Mehta NA, Cicalese MP, Casiraghi M, Boelens JJ, Del Carro U, Dow DJ, Schmidt M, Assanelli A, Neduva V, Di Serio C, Stupka E, Gardner J, von Kalle C, Bordignon C, Ciceri F, Rovelli A, Roncarolo MG, Aiuti A, Sessa M, Naldini L. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science. 2013 Aug 23;341(6148):1233158. doi: 10.1126/science.1233158. Epub 2013 Jul 11.
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PubMed Identifier
20979441
Citation
Frittoli MC, Biral E, Cappelli B, Zambelli M, Roncarolo MG, Ferrari G, Ciceri F, Marktel S. Bone marrow as a source of hematopoietic stem cells for human gene therapy of beta-thalassemia. Hum Gene Ther. 2011 Apr;22(4):507-13. doi: 10.1089/hum.2010.045. Epub 2011 Mar 4.
Results Reference
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PubMed Identifier
18650378
Citation
Miccio A, Cesari R, Lotti F, Rossi C, Sanvito F, Ponzoni M, Routledge SJ, Chow CM, Antoniou MN, Ferrari G. In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia. Proc Natl Acad Sci U S A. 2008 Jul 29;105(30):10547-52. doi: 10.1073/pnas.0711666105. Epub 2008 Jul 23.
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PubMed Identifier
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Citation
Montini E, Cesana D, Schmidt M, Sanvito F, Ponzoni M, Bartholomae C, Sergi Sergi L, Benedicenti F, Ambrosi A, Di Serio C, Doglioni C, von Kalle C, Naldini L. Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol. 2006 Jun;24(6):687-96. doi: 10.1038/nbt1216. Epub 2006 May 28.
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PubMed Identifier
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Citation
Roselli EA, Mezzadra R, Frittoli MC, Maruggi G, Biral E, Mavilio F, Mastropietro F, Amato A, Tonon G, Refaldi C, Cappellini MD, Andreani M, Lucarelli G, Roncarolo MG, Marktel S, Ferrari G. Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients. EMBO Mol Med. 2010 Aug;2(8):315-28. doi: 10.1002/emmm.201000083.
Results Reference
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Gene Therapy for Transfusion Dependent Beta-thalassemia

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