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PubPharm (252)
1
Exagamglogene Autotemcel for Severe Sickle Cell Disease
enthalten in:
The New England journal of medicine
| 2024
von
Frangoul, H.
|
Locatelli, F.
|
Sharma, A.
| +26
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2
Ovarian tissue cryopreservation for fertility preservation before hematopoietic stem cell transplantation in patients with sickle cell disease : safety, ovarian function follow-up, and results of ovarian tissue transplantation
enthalten in:
Journal of assisted reproduction and genetics
| 2024
von
Missontsa, M.
|
Bernaudin, F.
|
Fortin, A.
| +22
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3
Umbilical Cord Blood Transplantation for Fanconi Anemia With a Special Focus on Late Complications : a Study on Behalf of Eurocord and SAAWP-EBMT
enthalten in:
Transplantation and cellular therapy
| 2024
von
Rafii, H.
|
Volt, F.
|
Bierings, M.
| +25
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4
The difficult translational pathway from animal models to patients
enthalten in:
Cell stem cell
| 2024
von
Cavazzana, M.
|
Miccio, A.
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5
Method for generating T cells progenitors
enthalten in:
Europäisches Patentamt
| 2024
von
ANDRE ISABELLE
|
CAVAZZANA MARINA
|
MA KUIYING
| +3
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6
Autologous gene therapy for hemoglobinopathies : From bench to patient's bedside
enthalten in:
Molecular therapy : the journal of the American Society of Gene Therapy
| 2024
von
Locatelli, F.
|
Cavazzana, M.
|
Frangoul, H.
| +3
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7
Ovarian tissue cryopreservation for fertility preservation before hematopoietic stem cell transplantation in patients with sickle cell disease: safety, ovarian function follow-up, and results of ovarian tissue transplantation
enthalten in:
Journal of assisted reproduction and genetics
| 2024
von
Missontsa, M.
|
Bernaudin, F.
|
Fortin, A.
| +22
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8
Successful treatment of severe MSUD in Bckdhb-/- mice with neonatal AAV gene therapy
enthalten in:
Journal of inherited metabolic disease
| 2024
von
Pontoizeau, C.
|
Gaborit, C.
|
Tual, N.
| +13
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9
Successful treatment of severe MSUD in Bckdhb−/− mice with neonatal AAV gene therapy
enthalten in:
Journal of Inherited Metabolic Disease
| 2024
von
Pontoizeau, C.
|
Gaborit, C.
|
Tual, N.
| +13
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10
Rescuing the cytolytic function of APDS1 patient T cells via TALEN-mediated PIK3CD gene correction
enthalten in:
Molecular therapy. Methods & clinical development
| 2023
von
Poggi, L.
|
Chentout, L.
|
Lizot, S.
| +10
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bio
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gene therapy
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10
beta-Globins
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sickle cell disease
7
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6
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EC 2.7.1.137
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