Popping the Bubble: Promising Results From a Phase I-II Lentiviral Gene Therapy Trial for X-SCID

The Hematologist ◽

10.1182/hem.v16.4.9661 ◽

2019 ◽

Vol 16 (4) ◽

Author(s):

David T. Teachey ◽

Danielle E. Arnold

Keyword(s):

Gene Therapy ◽

Phase I ◽

Gene Therapy Trial ◽

Therapy Trial ◽

Lentiviral Gene Therapy

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Patient pre-selection outcomes for the European GENEGRAFT ex vivo phase I/II gene therapy trial for recessive dystrophic epidermolysis bullosa.

10.26226/morressier.595a9c56d462b80296c9fadf ◽

2017 ◽

Author(s):

Su Lwin

Keyword(s):

Gene Therapy ◽

Phase I ◽

Epidermolysis Bullosa ◽

Ex Vivo ◽

Dystrophic Epidermolysis Bullosa ◽

Gene Therapy Trial ◽

Recessive Dystrophic Epidermolysis Bullosa ◽

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A phase i hyperthermia- induced interleukin- 12 gene- therapy trial in a spontaneously arising feline soft tissue sarcoma model

International Journal of Radiation Oncology*Biology*Physics ◽

10.1016/j.ijrobp.2004.07.567 ◽

2004 ◽

Vol 60 (1) ◽

pp. S577

Author(s):

F. Siddiqui ◽

P.R. Avery ◽

S.M. LaRue ◽

M.L. Hauck ◽

M.W. Dewhirst ◽

...

Keyword(s):

Gene Therapy ◽

Soft Tissue ◽

Soft Tissue Sarcoma ◽

Phase I ◽

Interleukin 12 ◽

Gene Therapy Trial ◽

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EBGene trial: patient preselection outcomes for the European GENEGRAFT ex vivo phase I/II gene therapy trial for recessive dystrophic epidermolysis bullosa

British Journal of Dermatology ◽

10.1111/bjd.18559 ◽

2019 ◽

Vol 182 (3) ◽

pp. 794-797 ◽

Author(s):

S. Gaucher ◽

S.M. Lwin ◽

M. Titeux ◽

A. Abdul‐Wahab ◽

N. Pironon ◽

...

Keyword(s):

Gene Therapy ◽

Phase I ◽

Epidermolysis Bullosa ◽

Ex Vivo ◽

Dystrophic Epidermolysis Bullosa ◽

Gene Therapy Trial ◽

Recessive Dystrophic Epidermolysis Bullosa ◽

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A phase i hyperthermia- induced interleukin- 12 gene- therapy trial in a spontaneously arising feline soft tissue sarcoma model

International Journal of Radiation Oncology*Biology*Physics ◽

10.1016/s0360-3016(04)01871-1 ◽

2004 ◽

Vol 60 ◽

pp. S577-S577

Author(s):

F SIDDIQUI ◽

P AVERY ◽

S LARUE ◽

M HAUCK ◽

M DEWHIRST ◽

...

Keyword(s):

Gene Therapy ◽

Soft Tissue ◽

Soft Tissue Sarcoma ◽

Phase I ◽

Interleukin 12 ◽

Gene Therapy Trial ◽

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Participant perspectives on a phase I/II ocular gene therapy trial (NCT02077361)

Ophthalmic Genetics ◽

10.1080/13816810.2019.1630843 ◽

2019 ◽

Vol 40 (3) ◽

pp. 276-281

Author(s):

Stephanie P. Brooks ◽

Shelly Benjaminy ◽

Tania Bubela

Keyword(s):

Gene Therapy ◽

Phase I ◽

Gene Therapy Trial ◽

Therapy Trial ◽

Participant Perspectives

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A phase I/II gene therapy trial in metastatic melanoma patients

Journal of the American College of Surgeons ◽

10.1016/j.jamcollsurg.2006.05.217 ◽

2006 ◽

Vol 203 (3) ◽

pp. S83

Author(s):

Michel Adamina ◽

Walter P. Weber ◽

Rachel Rosenthal ◽

Reto Schumacher ◽

Paul Zajac ◽

...

Keyword(s):

Gene Therapy ◽

Phase I ◽

Metastatic Melanoma ◽

Gene Therapy Trial ◽

Melanoma Patients ◽

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227 Patient pre-selection outcomes for the European GENEGRAFT ex vivo phase I/II gene therapy trial for recessive dystrophic epidermolysis bullosa

Journal of Investigative Dermatology ◽

10.1016/j.jid.2017.07.224 ◽

2017 ◽

Vol 137 (10) ◽

pp. S231

Author(s):

S.M. Lwin ◽

S. Gaucher ◽

M. Titeux ◽

A. Abdul-Wahab ◽

N. Pironon ◽

...

Keyword(s):

Gene Therapy ◽

Phase I ◽

Epidermolysis Bullosa ◽

Ex Vivo ◽

Dystrophic Epidermolysis Bullosa ◽

Gene Therapy Trial ◽

Recessive Dystrophic Epidermolysis Bullosa ◽

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Phase I/II Gene Therapy Trial of Fanconi Anemia Patients with a New Orphan Drug Consisting of a Lentiviral Vector Carrying the FANCA Gene: A Coordinated International Action (EuroFancolen)

Human Gene Therapy Clinical Development ◽

10.1089/humc.2015.2522 ◽

2015 ◽

Vol 26 (2) ◽

pp. 81-82 ◽

Keyword(s):

Gene Therapy ◽

Phase I ◽

Orphan Drug ◽

Fanconi Anemia ◽

Lentiviral Vector ◽

Gene Therapy Trial ◽

International Action ◽

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Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial

Molecular Therapy ◽

10.1016/j.ymthe.2021.02.013 ◽

2021 ◽

Author(s):

Alaknanda Mishra ◽

Camasamudram Vijayasarathy ◽

Catherine A. Cukras ◽

Henry E. Wiley ◽

H. Nida Sen ◽

...

Keyword(s):

Gene Therapy ◽

Phase I ◽

Immune Function ◽

Gene Therapy Trial ◽

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Design, Baseline Characteristics, and 2-Year Follow-up from The MPS IIIA Natural History Study Used as Control Group in Gene Therapy Trial

10.1055/s-0039-1685419 ◽

2019 ◽

Author(s):

B. Heron ◽

A. Chakrapani ◽

P. Drevot ◽

R. Giugliani ◽

N. Muschol ◽

...

Keyword(s):

Gene Therapy ◽

Natural History ◽

Control Group ◽

Natural History Study ◽

Gene Therapy Trial ◽

Baseline Characteristics ◽

Therapy Trial ◽

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