PTD-mediated delivery of α-globin chain into Κ-562 erythroleukemia cells and α-thalassemic (HBH) patients’ RBCs ex vivo in the frame of Protein Replacement Therapy

In vivo biodistribution study of TAT-L-Sco2 fusion protein, developed as protein therapeutic for mitochondrial disorders attributed to SCO2 mutations

(2020) Georgios C. Kaiafas et al.   Molecular Genetics and Metabolism Reports

Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges

(2019) Valentina Ghiaccio et al.   Molecular Diagnosis & Therapy

Gene therapy of hemoglobinopathies: progress and future challenges

(2019) Yasuhiro Ikawa et al.   HUMAN MOLECULAR GENETICS

Genetic variation influencing hemoglobin levels and risk for anemia across populations

(2019) Paloma K. Barrera‐Reyes et al.   Annals of the New York Academy of Sciences

Molecular basis of α-thalassemia

(2018) Samaneh Farashi et al.   BLOOD CELLS MOLECULES AND DISEASES

Modified translationally controlled tumor protein-derived protein transduction domain enhances nasal delivery of exendin-4 as shown with insulin

(2018) Hae-Duck Bae et al.   DRUG DELIVERY

Production and Transduction of a Human Recombinant β-Globin Chain into Proerythroid K-562 Cells To Replace Missing Endogenous β-Globin

(2018) Lefkothea C. Papadopoulou et al.   MOLECULAR PHARMACEUTICS

In vitro and in vivo delivery of therapeutic proteins using cell penetrating peptides

(2017) Azam Bolhassani et al.   PEPTIDES

Cell-Penetrating Peptides: From Basic Research to Clinics

(2017) Giulia Guidotti et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Favorable outcomes afterin uterotransfusion in fetuses with alpha thalassemia major: a case series and review of the literature

(2016) Emily M. Kreger et al.   PRENATAL DIAGNOSIS

Gene therapy for hemoglobinopathies: progress and challenges

(2013) Alisa Dong et al.   Translational Research

Clinical Manifestations of  -Thalassemia

(2013) E. P. Vichinsky   Cold Spring Harbor Perspectives in Medicine

Protein transduction domain delivery of therapeutic macromolecules

(2011) Arjen van den Berg et al.   CURRENT OPINION IN BIOTECHNOLOGY

Transduction of Human Recombinant Proteins into Mitochondria as a Protein Therapeutic Approach for Mitochondrial Disorders

(2011) Lefkothea C. Papadopoulou et al.   PHARMACEUTICAL RESEARCH

Happy birthday cell penetrating peptides: Already 20 years

(2010) Robert Brasseur et al.   BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES

Intracellular delivery of full length recombinant human mitochondrial L-Sco2 protein into the mitochondria of permanent cell lines and SCO2 deficient patient's primary cells

(2010) Parthena F. Foltopoulou et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Protein transport in human cells mediated by covalently and noncovalently conjugated arginine-rich intracellular delivery peptides

(2009) Jia-Wei Hu et al.   PEPTIDES

siRNA delivery using peptide transduction domains

(2009) Akiko Eguchi et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Hb H disease: clinical course and disease modifiers

(2009) S. Fucharoen et al.   Hematology-American Society of Hematology Education Program