FDA-approved oligonucleotide therapies in 2017

CA Stein, D Castanotto - Molecular Therapy, 2017 - cell.com
Oligonucleotides (oligos) have been under clinical development for approximately the past
30 years, beginning with antisense oligonucleotides (ASOs) and apatmers and followed …

[HTML][HTML] A general practitioner's guide to hematopoietic stem-cell transplantation

A Bazinet, G Popradi - Current Oncology, 2019 - ncbi.nlm.nih.gov
Hematopoietic stem-cell transplantation (hsct) is a medical procedure that consists of
infusing stem cells after a short course of chemotherapy or radiotherapy, or both. It can be …

[HTML][HTML] CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia

H Frangoul, D Altshuler, MD Cappellini… - … England Journal of …, 2021 - Mass Medical Soc
Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe
monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is …

[HTML][HTML] Risk factors for development of and progression of hepatic veno-occlusive disease/sinusoidal obstruction syndrome

S Corbacioglu, EJ Jabbour, M Mohty - Biology of Blood and Marrow …, 2019 - Elsevier
Veno-occlusive disease, also known as sinusoidal obstruction syndrome (VOD/SOS), is a
potentially life-threatening complication of allogeneic or autologous hematopoietic stem cell …

Diagnosis and treatment of VOD/SOS after allogeneic hematopoietic stem cell transplantation

F Bonifazi, F Barbato, F Ravaioli, M Sessa… - Frontiers in …, 2020 - frontiersin.org
Hepatic veno-occlusive disease (VOD) or sinusoidal obstruction syndrome (SOS) is a rare
complication characterized by hepatomegaly, right-upper quadrant pain, jaundice, and …

Hepatic adverse event profile of inotuzumab ozogamicin in adult patients with relapsed or refractory acute lymphoblastic leukaemia: results from the open-label …

HM Kantarjian, DJ DeAngelo, AS Advani… - The Lancet …, 2017 - thelancet.com
Summary Background The INO-VATE study demonstrated efficacy and safety of inotuzumab
ozogamicin versus standard care in adults with relapsed or refractory B-cell acute …

Inotuzumab ozogamicin in adults with relapsed or refractory CD22-positive acute lymphoblastic leukemia: a phase 1/2 study

DJ DeAngelo, W Stock, AS Stein, A Shustov… - Blood …, 2017 - ashpublications.org
This study evaluated the safety, antitumor activity, pharmacokinetics, and
pharmacodynamics of inotuzumab ozogamicin (InO) for CD22-positive relapsed/refractory …

Management of important adverse events associated with inotuzumab ozogamicin: expert panel review

P Kebriaei, C Cutler, M De Lima, S Giralt… - Bone marrow …, 2018 - nature.com
Inotuzumab ozogamicin (InO), a humanized anti-CD22 monoclonal antibody conjugated to
the cytotoxic antibiotic agent calicheamicin, has demonstrated efficacy in the phase 3 INO …

Modified diagnostic criteria, grading classification and newly elucidated pathophysiology of hepatic SOS/VOD after haematopoietic cell transplantation

MS Cairo, KR Cooke, HM Lazarus… - British journal of …, 2020 - Wiley Online Library
Sinusoidal obstruction syndrome (SOS), previously known as hepatic veno‐occlusive
disease (VOD), remains a multi‐organ system complication following haematopoietic cell …

Endothelial cell function and endothelial‐related disorders following haematopoietic cell transplantation

GC Hildebrandt, N Chao - British Journal of Haematology, 2020 - Wiley Online Library
Use of haematopoietic cell transplantation (HCT) in the treatment of haematologic and
neoplastic diseases may lead to life‐threatening complications that cause substantial …