Results 191 to 200 of about 1,599,857 (245)

Molecular Glue Degraders in Early Development for Cancer Therapy

Medicinal Research Reviews, EarlyView.
ABSTRACT Molecular glue degraders are an emerging class of small molecule allosteric modulators that induce or stabilize protein‐protein interactions, enabling targeted degradation of previously intractable proteins. By redirecting E3 ligases to recognize neosubstrates, proteins that are not typically recognized by a specific E3 ubiquitin ligase, they ...
E. Sila Ozdemir, Hyunbum Jang, Ozlem Keskin, Attila Gursoy, Ruth Nussinov   +4 more
wiley   +1 more source

Early venetoclax dose escalation at minimal residual disease progression may benefit selected acute myeloid leukaemia patients. [PDF]

Br J Haematol
Kytölä S, Partanen A, Kuusisto MEL, Koskela S, Holopainen A, Vänttinen I, Ruokoranta T, Ettala P, Rimpiläinen J, Siitonen T, Pyörälä M, Kuusanmäki H, Kontro M.   +12 more
europepmc   +1 more source

Assessment of Lectin Staining Biomarkers for GNE Myopathy Gene Therapy

Muscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims GNE myopathy (GNEM) is a rare, autosomal recessive disorder caused by mutations in the UDP‐N‐acetylglucosamine (GlcNAc) 2‐epimerase/N‐acetylmannosamine (ManNAc) kinase (GNE) gene, which encodes a required enzyme for sialic acid (SA) biosynthesis.
Kristina M. Sattler, Brian J. Paleo, Mackenzie N. Roth, Kara E. Bradley, Megan N. Winkler, Wakako Yoshioka, Ichizo Nishino, Christoph Lepper, Kelly E. Crowe   +8 more
wiley   +1 more source

In Utero HSC Transplantation for Sickle Cell Disease: A Potential Therapeutic Approach That Overcomes Complications of Current Therapies

Prenatal Diagnosis, EarlyView.
ABSTRACT Sickle cell disease (SCD) affects millions worldwide but has limited treatment options, most of which carry significant side effects. At present, the only curative treatment for SCD is allogeneic or gene‐modified autologous hematopoietic stem cell (HSC) transplantation (Tx).
Oluwaseun O. Babatunde, Madeleine G. Bibby, Anthony Atala, Graça Almeida‐Porada, Christopher D. Porada   +4 more
wiley   +1 more source

Quality of life and symptoms in acute myeloid leukaemia with early palliative care: real-world observational study. [PDF]

BMJ Support Palliat Care
Potenza L, Giusti D, Borelli E, Colaci E, Morandi F, Cuoghi Costantini R, Del Giovane C, Forghieri F, Morselli M, Bettelli F, Pioli V, Cuoghi A, Bresciani P, Messerotti A, Gilioli A, Marasca R, Cassanelli L, Parisotto A, Cretì F, Mucciarini C, Bigi S, D'Amico R, Efficace F, Odejide O, Bruera E, Zimmermann C, Luppi M, Bandieri E.   +27 more
europepmc   +1 more source

Guillain–Barre Syndrome With Cytomegalovirus Infection After Allogeneic Hematopoietic Stem Cell Transplantation

The Kaohsiung Journal of Medical Sciences, EarlyView.
Chin‐Mu Hsu, Ping‐Hsuan Lin, Chia‐Hsuan Chiu, Hui‐Hua Hsiao   +3 more
wiley   +1 more source

Structural Variation Sequencing of 26 Amniotic Fluid Samples With Partial Gene Duplications and Postnatal Follow‐Up of the Fetuses

Prenatal Diagnosis, EarlyView.
ABSTRACT Objectives Partial gene duplications (PGDups) are a significant contributor to genetic disease. The precise genomic location and structure of PGDups are often unresolved using conventional methods, so prenatal diagnosis for PGDups is challenging, especially without ultrasound abnormalities.
Shengfang Qin, Xueyan Wang, Mengjia Yan, Yuxia He, Zhuo Zhang, Yan Yin, Jin Wang, Beibei Guo, Jing Wang, Na Xi, Zhaokai Li   +10 more
wiley   +1 more source

Outcomes of patients with de novo and secondary acute myeloid leukaemia treated with front-line hypomethylating agents and venetoclax: A retrospective Italian study. [PDF]

Br J Haematol
Tanasi I, Tamellini E, Marchetti E, De Bellis E, Ancora C, Angotzi F, Facchinelli D, Capraro F, Battaglia G, Sperotto A, Gottardi M, Leoncin M, Krampera M, Tiribelli M, Gurrieri C.   +14 more
europepmc   +1 more source

Spectrum of actionable genetic variants in acute myeloid leukaemia patients from Pakistan: a study based on next-generation sequencing. [PDF]

Ecancermedicalscience
Khurram S, Basit AA, Qureshi MA.
europepmc   +1 more source

Correction to "A Delphi Consensus on Optimising the Care Pathway for Adult Patients With Acute Myeloid Leukaemia (AML): Strategies to Enhance Transplant Accessibility and Feasibility in the United Kingdom". [PDF]

EJHaem
europepmc   +1 more source