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Protein replacement therapy is a medical treatment that supplements or replaces a protein in patients in whom that particular protein is deficient or
Protein_replacement_therapy
Medical treatment
Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. Usually, this is done by giving
Enzyme_replacement_therapy
Next-generation mRNA vaccine/therapeutic
This sequence encodes the protein of interest, used as an antigen in the case of vaccines or for protein replacement therapies. The gene of interest replaces
Self-amplifying_RNA
Type of gender-affirming medical treatment
Feminizing hormone therapy, also known as transfeminine hormone therapy, is a form of gender-affirming care and a gender-affirming hormone therapy to change the
Feminizing_hormone_therapy
Hungarian-American biochemist (born 1955)
particularly in vitro-transcribed messenger RNA (mRNA) for protein replacement therapy. Karikó laid the scientific groundwork for mRNA vaccines, overcoming
Katalin_Karikó
Change of a linear protein chain to a 3D structure
where loss of function is the origin of the disorder. While protein replacement therapy has historically been used to correct the latter disorders, an
Protein_folding
Medical condition
complications, as with any protein replacement therapy. Concomitant warfarin therapy in subjects with congenital protein C deficiency is associated with
Purpura_fulminans
Special form of in vitro fertilisation
Mitochondrial replacement therapy (MRT), sometimes called mitochondrial donation, is the replacement of mitochondria in one or more cells to prevent or
Mitochondrial replacement therapy
Mitochondrial_replacement_therapy
epigenetic therapy by proteins: protein therapy (but many drugs are proteins despite not being called protein therapy) by enzymes: enzyme replacement therapy by
List_of_therapies
particularly in vitro-transcribed messenger RNA (mRNA) for protein replacement therapy. Karikó laid the scientific groundwork for mRNA vaccines and
List of awards and honors received by Katalin Karikó
List_of_awards_and_honors_received_by_Katalin_Karikó
Blood vessel formation, when new vessels emerge from existing vessels
three main categories: gene therapy, targeting genes of interest for amplification or inhibition; protein replacement therapy, which primarily manipulates
Angiogenesis
Genetic disorder group
symptoms. Treatment of the disease currently involves enzyme replacement therapy and gene therapy is a possibility for the future, a solution which may cure
Autophagic_vacuolar_myopathy
American biotech firm
March 2017). "Systemic delivery of factor IX messenger RNA for protein replacement therapy". PNAS. 114 (10): E1941–E1950. Bibcode:2017PNAS..114E1941R. doi:10
Arcturus_Therapeutics
Type of fluid replacement used to prevent and treat dehydration
Oral rehydration therapy (ORT) is a type of fluid replacement used to prevent and treat dehydration, especially due to diarrhea. It involves drinking water
Oral_rehydration_therapy
Drug delivery method
a wide range of genetic disorders, enabling advancements in protein replacement therapy and gene editing, as this strategy allows for gene editing without
Selective_organ_targeting
Medical technology
mutation and thereby produces a functional protein. This strategy is referred to as gene replacement therapy and could be employed to treat inherited retinal
Gene_therapy
their research on in vitro-transcribed messenger RNA (mRNA) for protein replacement therapy. biochemist Tibor Radó (1895–1965) Hungarian mathematician; research
List of University of Szeged people
List_of_University_of_Szeged_people
Use of stem cells to treat or prevent a disease or condition
Stem-cell therapy uses stem cells to treat or prevent a disease or condition. As of 2024[update], the only FDA-approved therapy using stem cells is hematopoietic
Stem-cell_therapy
Type of hormone therapy
we suggest the use of two tests before making this diagnosis." GH replacement therapy can provide a number of measurable benefits to GH-deficient adults
Growth_hormone_therapy
Surgical procedure
corrected by osteotomy. Physical therapy has been shown to improve function and may delay or prevent the need for knee replacement. Pain is often noted when
Knee_replacement
Medical intervention
chondrogenesis at the site of the contained lesion. Meniscal cartilage replacement therapy Meniscus transplant Spheroids of human autologous matrix-associated
Knee cartilage replacement therapy
Knee_cartilage_replacement_therapy
Medication administered into a vein
Intravenous therapy (abbreviated as IV therapy) is a medical process that administers fluids, medications and nutrients directly into a person's vein.
Intravenous_therapy
Local or general use of low temperatures in medical therapy
Cryotherapy, sometimes known as cold therapy, is the local or general use of low temperatures in medical therapy. Cryotherapy can be used in many ways
Cryotherapy
Genetic disorder affecting mostly the lungs
defects in the CFTR protein rather than only treating the downstream symptoms of cystic fibrosis. Eligibility for CFTR modulator therapy depends on a person’s
Cystic_fibrosis
Cancer of plasma cells
myeloma is diagnosed based on blood or urine tests finding abnormal antibody proteins (often using electrophoretic techniques revealing the presence of a monoclonal
Multiple_myeloma
Buildup of metabolic waste due to kidney dysfunction
for protein toxicity can include renal replacement therapies like hemodialysis and hemofiltration. Lifestyle modifications like a diet low in protein, decreased
Protein_toxicity
Species of annelid worm
the end of the 19th century. Medicinal leech therapy (also referred to as Hirudotherapy or Hirudin therapy) made an international comeback in the 1970s
Hirudo_medicinalis
Drug delivery method
One of the earliest proposed applications of EBDDS was in enzyme replacement therapy for Gaucher disease, in which lipids accumulate in spleen macrophages;
Erythrocyte-based drug delivery
Erythrocyte-based_drug_delivery
American NGO leader and patient advocate, 1949–2017
Health, which ran through 1995 and ended in the development of a protein replacement therapy treatment. During the study, Walsh and others formed support
John_W._Walsh
Drug that acts as a protein chaperone
by use of pharmacoperones. Autochaperone Enzyme replacement therapy "Drugs to fix "misfolded" proteins could cure a range of diseases". Gizmag.com. 10
Pharmacological_chaperone
Amylase, lipase, protease and lactase mixture
taken by mouth. The treatment is also known as pancreatic enzyme replacement therapy or PERT. Common side effects include vomiting, abdominal pain, constipation
Pancreatic enzymes (medication)
Pancreatic_enzymes_(medication)
Investigational Parkinson's disease drugs
therapy) – dopaminergic cell replacement [62] CT1-DAP001/DSP-1083 (allo iPS cell-derived dopamine neural progenitor) – dopaminergic cell replacement [63]
List of investigational Parkinson's disease drugs
List_of_investigational_Parkinson's_disease_drugs
Rare, severe disease of lysosomal storage
in certain populations. Researchers are looking at gene therapy or enzyme replacement therapy as possible treatments. Tay–Sachs disease is typically first
Tay–Sachs_disease
Medical condition
illegally. People with opioid use disorder are often treated with opioid replacement therapy using methadone or buprenorphine. Such treatment reduces the risk
Opioid_use_disorder
Medical administration of exogenous surfactant
For more details, see Pulmonary surfactant. Surfactant therapy, or surfactant replacement therapy, is used in situations where there is not sufficient fluid
Surfactant_therapy
Medical study concerned with the kidneys
"kidney replacement therapy". Other experts advocate preserving the use of renal and nephro- including in "nephrology" and "renal replacement therapy". Nephrology
Nephrology
Treatment for blindness
Retinal gene therapy holds a promise in treating different forms of non-inherited and inherited blindness. In 2008, three independent research groups
Gene therapy of the human retina
Gene_therapy_of_the_human_retina
American geneticist
enzyme replacement therapy (ERT) and the chaperone therapy for Fabry disease, ERT for Niemann–Pick disease type B, and the RNA Interference Therapy for the
Robert_J._Desnick
risk factors for the disease. Gene augmentation, gene replacement, and novel transgene gene therapy strategies for the potential medical management of osteoarthritis
Gene therapy for osteoarthritis
Gene_therapy_for_osteoarthritis
Protein-coding gene in the species Homo sapiens
morphogenetic protein 7 or BMP7 (also known as osteogenic protein-1 or OP-1) is a protein that in humans is encoded by the BMP7 gene. The protein encoded by
Bone_morphogenetic_protein_7
Gene therapy medication
is a gene therapy used for the treatment of hemophilia B. Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists
Etranacogene_dezaparvovec
Disease where the kidneys fail to adequately filter waste products from the blood
glomerular filtration rate (GFR) of less than 15 or the need for renal replacement therapy. It is also equivalent to stage 5 chronic kidney disease. Treatment
Kidney_failure
Abnormal kidney structure or gradual loss of kidney function
Preparation for kidney replacement therapy. Stage 5: Established kidney failure (GFR <15 mL/min/1.73 m2), permanent kidney replacement therapy, or end-stage kidney
Chronic_kidney_disease
X-linked recessive genetic condition
dose of idursulfase than the intravenous formulation used in enzyme replacement therapy infusions, in hopes of preventing the cognitive decline associated
Hunter_syndrome
Inherited neurodegenerative disorder
micro-RNA to silence the gene leading to a lowering of huntingtin protein expression. The therapy is delivered directly into the deep brain structures of the
Huntington's_disease
German oncologist and immunologist (born 1965)
immunotherapies, as vaccines against infectious diseases, and as protein replacement therapies for rare diseases. He holds a minority interest in the listed
Uğur_Şahin
Species of virus
AAV capsid protein VP1, which may be attractive targets for modification of the capsid if the virus is to be used as a vector for gene therapy. There are
Adeno-associated_virus
Vaccine protecting against tooth decay
Characterization of an Effector Strain of Streptococcus mutans for Replacement Therapy of Dental Caries". Infection and Immunity. 68 (2): 543–549. doi:10
Caries_vaccine
Chemical compound
are shipped to the late endosomes via vesicular transport. Enzyme replacement therapy (ERT) for several lysosomal storage diseases relies on this pathway
Mannose_6-phosphate
Therapy for the treatment of Parkinson's disease
which they use a gene enzyme replacement therapy that can be used to increase the efficacy of the pharmacological L-dopa therapy by using AAV vectors. This
Gene therapy in Parkinson's disease
Gene_therapy_in_Parkinson's_disease
Medical condition
antimalarial drug therapy (e.g., artemisinin derivatives), fluid replacement (e.g., oral rehydration therapy), and if needed, renal replacement therapy. Malarial
Malarial_nephropathy
Medical condition
(August 1993). "Replacement therapy with a purified protein C concentrate during initiation of oral anticoagulation in severe protein C congenital deficiency"
Warfarin_necrosis
Condition involving impaired blood clotting ability
with medications or replacement therapy. In replacement therapy, the reduced or absent clotting factors are replaced with proteins derived from human blood
Coagulopathy
Cancer that originates in mammary glands
obesity, a lack of physical exercise, alcohol consumption, hormone replacement therapy during menopause, ionizing radiation, an early age at first menstruation
Breast_cancer
overshadowed any beneficial outcomes. On the other hand, Estrogen Replacement Therapy has shown some positive effects with postmenopausal women. Estrogen
Estrogen and neurodegenerative diseases
Estrogen_and_neurodegenerative_diseases
Genetic disease involving blood clotting
restore the clotting factor IX (FIX) protein, normal levels of the protein were observed with low doses of the therapy but immunosuppression was necessitated
Haemophilia
Group of neural structures responsible for motivation and desire
intracellular cascade activated by D1 receptors involves the recruitment of protein kinase A, and through resulting phosphorylation of DARPP-32, the inhibition
Reward_system
Rare human genetic lysosomal storage disorder
organ damage, and therapies to improve symptoms and life expectancy once organ damage has already occurred. Enzyme replacement therapy is designed to provide
Fabry_disease
Thyroid hormone medication
stomach about half an hour to an hour before meals. As such, thyroid replacement therapy is usually taken 30 minutes prior to eating in the morning. For patients
Levothyroxine
(saRNA) offer even greater promise of lower dose vaccines and protein replacement therapies. While saRNA shows promise in preclinical and clinical studies
Mark_W._Grinstaff
Swiss pharmaceutical company
underlying principle of Octapharma's R&D is the development of therapies based on human proteins either purified from human plasma or produced by recombinant
Octapharma
Type of autoimmune arthritis
those miRNAs after therapy, and paralleled the reduction of TNFα, interleukin (IL)-6, IL-17, rheumatoid factor (RF), and C-reactive protein (CRP). Many tools
Rheumatoid_arthritis
Medical condition
sometimes called "read-through therapy", translational skipping of the stop codon, resulting in a functional protein, can be induced by the introduction
Hypotrichosis with juvenile macular dystrophy
Hypotrichosis_with_juvenile_macular_dystrophy
Removal, treatment and return of blood plasma
as a therapy. This is also called cascade filtration. Exchange, a patient's blood plasma is removed, while blood products are given in replacement. This
Plasmapheresis
Type of muscular dystrophy
mutations or deletions in any of the 79 exons encoding the large dystrophin protein, which is essential for maintaining the muscle fibers' cell membrane integrity
Duchenne_muscular_dystrophy
Medical condition
reductase protein. SAAM hypothetically triggers this increase in the production of HMG-CoA reductase and associated abnormal processing of this protein in genetically
Statin-associated autoimmune myopathy
Statin-associated_autoimmune_myopathy
Substrate reduction therapy offers an approach to treatment of certain metabolic disorders, especially glycogen storage diseases and lysosomal storage
Substrate_reduction_therapy
Medical condition
outpatient program Methadone maintenance Smoking cessation Nicotine replacement therapy Tobacco cessation clinics in India Twelve-step program Support groups
Caffeine_dependence
Medical treatment
destroys the protective covering over the nerves. It is indicated for replacement therapy in adults and children in primary immunodeficiency syndromes such
Immunoglobulin_therapy
Medical condition
effectiveness of A1AT replacement therapy are not available. It is currently recommended that patients begin augmentation therapy only after the onset
Alpha-1 antitrypsin deficiency
Alpha-1_antitrypsin_deficiency
Sollpura (Liprotamase), a pancreatic enzyme replacement therapy (PERT). It assists in the breakdown of proteins into amino acids and polypeptides. Bromelain
Proteases (medical and related uses)
Proteases_(medical_and_related_uses)
Formation of bone tissue outside of the skeleton
success and efficacy of physical therapy (PT) and passive range of motion (PROM). In patients that had a total knee replacement, PT and PROM was found to be
Heterotopic_ossification
Chemical compound
fetus. Hypothyroid mothers should continue to take thyroid hormone replacement therapy throughout pregnancy to avoid adverse events. Breastmilk contains
Liothyronine
Medical condition
(Brineura) as the first specific treatment for NCL. It is enzyme replacement therapy manufactured through recombinant DNA technology. The active ingredient
Neuronal ceroid lipofuscinosis
Neuronal_ceroid_lipofuscinosis
Compulsion to engage in a non-substance related behavior
promote healthier behaviors. Because cognitive behavioral therapy is considered a short-term therapy, the number of sessions for treatment normally ranges
Behavioral_addiction
Other uses of Placenta
Injection from fresh human Placenta called Inj.Placentrex. 2 ml. Hormone replacement therapy containing estrogen, while protective against osteoporosis, has been
Alternative_uses_for_placenta
used for both research and medical applications, such as enzyme replacement therapy. Erythropoietin (EPO): Epogen from Amgen Granulocyte colony-stimulating
List_of_recombinant_proteins
Genetic multisystem copper-transport disease
stimulates metallothionein, a protein in gut cells that binds copper and prevents its absorption and transport to the liver. Zinc therapy is continued unless symptoms
Wilson's_disease
Genetic disorder which disrupts normal functioning of the olfactory and pituitary glands
only found in people who have undergone some form of testosterone replacement therapy. It is only normally discovered when testicular volume increases
Kallmann_syndrome
Medical condition
Franceschi, Marianna; Fuchs, Claudia; et al. (1 May 2018). "CDKL5 protein substitution therapy rescues neurological phenotypes of a mouse model of CDKL5 disorder"
CDKL5_deficiency_disorder
Powdered breakfast food
introduced in 1964. It is a powdered instant drink that is manufactured with protein, vitamins and minerals and sugar. It is typically prepared with milk, and
Instant_breakfast
Rare genetic disorder
oligonucleotides therapy might be promising to specifically increase productive SLC6A1 mRNA and consequently restore levels of GAT1 protein Observational
SLC6A1 epileptic encephalopathy
SLC6A1_epileptic_encephalopathy
Gene therapy medication
abeparvovec, sold under the brand name Zolgensma among others, is a gene therapy used to treat spinal muscular atrophy, a disease causing muscle function
Onasemnogene_abeparvovec
Hormones produced by the adrenal cortex
to supply the hormones it produces and therefore requires hormone replacement therapy. Addison's disease is an autoimmune disorder that affects the adrenal
Adrenocortical_hormone
Protein created by joining other proteins into a single polypeptide
Fusion proteins or chimeric proteins (literally, made of parts from different sources) are proteins created through the joining of two or more genes that
Fusion_protein
Swedish pharmaceutical company
regarding its Kiobrina phase II program, an investigational enzyme replacement therapy to improve growth in preterm infants who receive pasteurized breast
Swedish_Orphan_Biovitrum
Form of RNA splicing used to cause cells to "skip" over faulty sections of genetic code
create prematurely truncated proteins that are unable to perform their job. Such mutations lead to muscle fiber damage, replacement of muscle tissue by fat
Exon_skipping
Genetic gastrointestinal disorder
diagnosis and aggressive salt replacement therapy result in normal growth and development, and generally good outcomes. Replacement of NaCl and KCl has been
Congenital_chloride_diarrhea
Medical condition
but with lower abuse liability, could theoretically be used as replacement therapy in amphetamine dependence. However, the few studies that used amphetamine
Amphetamine_dependence
Medical condition
be continually monitored. The most common treatment for PPNAD is replacement therapy and bilateral laparoscopic adrenalectomy; the process by which both
Primary pigmented nodular adrenocortical disease
Primary_pigmented_nodular_adrenocortical_disease
Progressive neurodegenerative disease
administration required to administer some peptides and proteins to the brain. MicroRNAs are an emerging PD gene therapy platform that may serve as an alternative to
Parkinson's_disease
Insufficient production of thyroid hormones
hypothyroidism in children and adolescents and potential effects of replacement therapy: a review". European Journal of Endocrinology. 168 (1): R1–R11. doi:10
Hypothyroidism
Metabolic disorders affecting a cell's lysosomes
mostly symptomatic, although bone marrow transplantation and enzyme replacement therapy (ERT) have been tried with some success. ERT can minimize symptoms
Lysosomal_storage_disease
Diet with very or extremely low daily food energy consumption
elements, fatty acids, protein and electrolyte balance. Carbohydrates may be entirely absent, or substituted for a portion of the protein; this choice has important
Very-low-calorie_diet
Chemical compound
it useful for producing masculinization and suitable for androgen replacement therapy. Esterase enzymes break the ester bond in testosterone enanthate
Testosterone_enanthate
routes of estrogen replacement therapy on 24-hour growth hormone (GH) secretion, insulin-like growth factor I, and GH-binding protein in postmenopausal
Pharmacokinetics_of_estradiol
Lysosomal storage disease
systemic complications. These complications often develop despite renal replacement therapy: Ocular: corneal cystine crystal deposition causes photophobia, eye
Cystinosis
Medical condition
standard treatment for Bamforth–Lazarus syndrome is thyroid hormone replacement therapy. Most commonly, synthetic thyroid hormone called levothyroxine (LT4)
Bamforth–Lazarus_syndrome
Pharmaceutical drug
which now prevent nearly all forms of disease transmission during replacement therapy.[citation needed] von Willebrand Factor/Coagulation Factor VIII Complex
Factor_VIII_(medication)
Genetic X-linked recessive bleeding disorder
restore the clotting factor IX (FIX) protein, normal levels of the protein were observed with low doses of the therapy but immunosuppression was necessitated
Haemophilia_B
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